Tuesday, February 4, 2014

New Treatment Options?

CUTAG of the Week: New Treatment Options?

Read this news link titled "Gene Therapy Shows Promise in Controlling HIV"

http://www.aidsmeds.com/articles/Sangamo_genetics_1667_24579.shtml

Based on this week's discussion on HIV, genetics, treatment, and, the news article above, do you think gene therapy has a promising future as a treatment for HIV/AIDS? Describe the strengths and limitations of your argument.    


OR choose one of the four following activities and share your response in the "Comment Section below":
1. You can share the new, 'surprising' information you learned
2. Ask a question that needs additional clarification,
3. Share a news link relevant to the discussion. 
4. Reply to another student's comment.

76 comments:

  1. Since we're talking about HIV in class, this is a great article that was published just a few days ago.

    http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0087487;jsessionid=45989588E197E4F67E74FC14365E554A#s5

    The article evaluates the medicinal properties of root extract from the native South African plant, Pelargonium sidoides (common name Geranium), in treating HIV-1, the most common strain of HIV in the world. This is a great article to read especially since we just recently discussed in class how the HIV virus infects your cells. If you read the article, you'll recognize a lot of familiar scientific terms such as CD4 cell surface receptors. The results presented in the article are very compelling and could potentially open up a whole new "herbal medicine" based field for HIV-1 treatment. This would have an incredible impact in medicine and diversify the types of treatments we would have to combat the HIV pandemic.

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    1. Nice!

      http://www.sciencedirect.com/science/article/pii/S0378874109002712

      This is so cool! I looked into this a bit further and it turns out that there is actually extensive research done on the topic of herbal anti-viral therapy for HIV-1. There are five extracts that look promising from the Geranium plants. It's great that there are plants that can inhibit the activity of HIV-1 as well as reducing the replication of the virus. However with all that said, the chemokine receptors were only inhibited HIV-1 activity by 33% in the instances of plant extraction. I think we would need something with a stronger power to really have a huge impact on the war against AIDS. It's also great that herbal medicine is being taken more seriously in modern medicine!

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  2. Today in class we discussed chemokines acting as messengers binding to receptors on T cells. Understanding this knowledge gives the option of gene therapy that prevents CCR5 from binding to T cells in turn keeping T cells inactive and unable to migrate to host cells. The article I found by Yves Pommier, Allison A. Johnson and Christophe Marchand discusses an alternative gene therapy of targeting the step of HIV infection where HIV intergrase integrates double strand DNA into host DNA. This gene therapy would act to inhibit intergrase from integrating the viral DNA into the DNA of the host cell rendering it inactive.

    http://www.discover.nci.nih.gov/pommier/2005.Pommier.nrd.pdf

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  3. I think gene therapy definitely has a promising future, though as with all things, I am sure it would need to be extensively researched to be rendered safe. This article was very interesting. In some of my previous classes, I have learned about some treatments that they already use for HIV – treatments such as reverse transcriptase inhibitors, protease inhibitors, and fusion inhibitors. Unfortunately, these are just treatments, not cures. Reverse transcriptase inhibitors and protease inhibitors only work after HIV has already infected the CD4 cell, because they affect some of the steps that occur after HIV enters the cell. All of these drugs can have serious side effects, and all come with the risk of the patient developing drug resistance.

    This new treatment is one that I had not heard of before, involving the manipulation of a person’s own genes in their blood to knock out the CCR5 expressing gene. I think this is a great idea, and would be able to work so that HIV would not even be able to bind to receptors on the cell, so would stop CD4 cells from being infected in the first place. Also, it would not be using a drug, so the patient would not develop drug resistance. It also seems to be more of a long-term plan, as these newly engineered cells would be able to resist HIV without having ongoing treatment.

    I hesitate to give this my full support just yet, but this is definitely in the right direction. Watching the rest of the video “Ghost in Your Genes,” I saw that DNA can be negatively affected simply by taking them out of their natural habitat, as in the in vitro fertilization example. By taking a patient’s blood out of their body, manipulating it, and then reinfusing the blood, there are a lot of steps that could carry the risk of negative outcomes. However, I do think that with further research on how to ensure the safety of this procedure, this could be a better option for HIV patients compared to drug therapy.

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  4. The future for gene therapy sounds promising, especially for those seeking treatment against HIV/AIDS. It is amazing that we can finally have a sense of control over HIV/AIDS, which used to be viewed as a dead end. From what I understood from the article, this gene therapy can inhibit viral growth. Though that is great news, it is important to remember this is not a cure. It is not to say all that work was done in vain; this can give people hope that even though they have HIV/AIDS, they can exert some type of control over it. While the growth of the disease is put on pause, more time will be granted to research ways to get rid of HIV/AIDS. I am worried that, given the adaptive nature of viruses, they will find a way around this gene therapy, and find a new way to infect a host cell. I have learned in classes that it only takes one virus to circumvent an obstacle, and once it learns how to do so, the virus can communicate with other viruses and share this information. I am hopeful that a process to not only inhibit viruses, but also to rid of them, will be discovered.

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  5. This article shares some very exciting news about the future of AIDS treatment. As genetics has proven to be a very difficult science to understand thus far, it is great to read about such incredible progress being made. Even still, this treatment is still in the very early stages; it has only been tried on 11 people, with only 3 of the subjects experiencing the complete desired results. Clearly researchers still have a long way to go, but their findings are quite promising. However, I am interested to know if there are any side effects associated with treatment, or with any gene therapies. I'm not really educated on the matter, but I would assume that altering one's DNA in some way would have some side effect. I look forward to learning about the possible side effects in the future!

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  6. I never really thought much about genetic therapy before this class because it's never been a topic I've looked into but this article shows that the future is promising for research in HIV treatments. Given the amount of people worldwide who have HIV/AIDS, a lot of research has been going on and it's exciting to see that there has been some progress in the genetic therapy from the article on the CCR5 receptor for a few people. It would be better if the therapy had worked on more than 3 people, but it shows that they are heading in the right direction. The researchers made sure to point out that "sustained functional control of HIV in the absence of ART is possible," showing that they also know there is a long way to go.

    I'm excited to learn more about genetic therapies for other major diseases as well as HIV. It's extremely interesting to think that genetics can play such an enormous role in many diseases along with environmental factors.

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  7. Even though we have yet to find cures for many diseases, like HIV/AIDS, it's really amazing how much progress has been made and how much we have done and discovered in science. Regarding the article, like Lindsay, I also thought it would have been better if the therapy worked on more than 3 of the participants. However, this is an extremely good start in dealing with HIV. Although this therapy does not fully cure HIV, I hope continuous research with this sort of therapy will prove to more effective in the future for those with HIV and hopefully one day we can find an absolute cure for HIV/AIDS.

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  8. Before reading the article posted above, I had no idea such genetic therapy for HIV/AIDS even existed. Thus, I found this article very interesting. I learned that you can genetically engineer cells, by removing the CCR5-expressing gene, and then reinfuse that cell back into the body. Although the treatment does look good so far in the sense that viral load was shown to decrease and increase CD4 cells, the study is too small and not far enough along to be considered reliable and valid. It is definitely exciting to think that gene therapy could help to cure HIV/AID once the research is completed.

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  9. So far, the gene therapy seems promising. However, there still has a lot be done. Very few individuals were used for the trials, and I think that more studies need to be done. While I think it looks promising and I’m hopeful, I remain cautious on how close to a HIV cure this therapy is. Researchers tend to overvalue their data and can be purposely misleading. But I'm optimistic and believe that we are always moving closer to finding a possible cure with all the new research that appears.
    This article has more updated information on this therapy: http://investor.sangamo.com/releasedetail.cfm?ReleaseID=803951

    A new HIV vaccine that allows HIV infected individuals to produce T-cells designed to fight HIV has recently appeared in the news. Clinical trials that are going involve over 1,000 patients are set to start in spring 2014.
    http://www.policymic.com/articles/78021/new-vaccine-may-eliminate-hiv-completely

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  10. I think that gene therapy is the correct avenue of targeting HIV infection because I think it will eventually grant us with the ability of focusing on HIV prevention rather than regulation. However, I think there are a few shortcomings with this study. First and foremost, those who are eligible for this treatment must have one non-functional version of the CCR5 receptor. In addition, this therapy would only be targeted against a certain versions of the retrovirus such as the "CCR5 tropic" strain.

    I can't help but wonder if we managed to intervene with T-cell development if we would be able to have T-cells already waiting in our nodes (like the thymus, lymphoid organs, etc) ready to respond if we ever came in contact with HIV. Nevertheless, to achieve this we would have to discover a way to prep the body against infection, possibly through vaccination. But, vaccination is another issue in itself because it has proven to be difficult to weaken the HIV virus enough to where it still stimulates an appropriate immune response. In addition to this proposition, I think it would also be beneficial to look into encouraging CCR5 receptor mutations that prevent the integration of HIV into the CD4T-cells. To accomplish this, I think more research on the integration of these mutations into those in which it does not naturally occur in would be necessary.

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  11. Even before reading the article, I thought that gene therapy is a promising technique to use for the treatment of HIV/AIDS. After reading the article, I still believe that it is. We as a society want to improve our lives and curing or treating diseases is high on our list. While the article highlights the cases of 11 people, only 7 actually had a positive response. This is promising! But it is necessary to follow these 7 participants for years to truly see if the gene therapy worked to effectively prove that HIV/AIDS can be controlled. Researchers at Brigham and Women’s Hospital hoped to successfully cure HIV/AIDS through bone marrow transplants, only to have the virus reappear 12 and 32 weeks after the end of antiviral therapy:
    http://www.cnn.com/2013/12/07/health/hiv-patients/
    I believe that gene therapy is important to study, because it has the potential to cure diseases and save lives. There are still technical setbacks that can hinder the true use of gene therapy, like finding better ways for genes to be targeted to certain cells. There are also health risks, like toxicity and cancer. There are ethical dilemmas (such as gene therapy on sex cells, which may allow for inheritance changes), regulations and laws, and societal expectations (which also may be linked with ethical standards). I think that we have enough guidelines and agencies in place to combat these risks and make it safe for eventual consumers. But we won’t know the value of gene therapy until we have the research and technology to use it effectively as treatment.

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  12. After reading the article posted, I wanted to know more and ended up finding this news article for 2011: http://www.nbcnews.com/id/41834106/ns/health-aids/t/gene-therapy-builds-hope-future-aids-cure/#.UvJishZCdUQ. This article talks about the beginning of the project in which the results were mentioned in the first article. I thought it was interesting to see kind of the before and after (or during) of the project from 2011 to 2013.

    One thing that was interesting in the article that I found was when it talked about how exactly the gene therapy happens. It says, "this is the first time researcher have permanently deleted a human gene and infused the altered cells back into patients. Other gene therapy attempts tried to add a gene or muffle the activity of one, and have not worked against HIV." I thought that it was interesting how many different ways the gene therapy could be provided and how to figure out which one works.

    Also in the article I posted above, it mentions that growth factors were also added to the newly formed T-cells before they were infused back into the patient. I found this to be very interesting and based on the results, it seems as though it worked to make more of the resistant T-cells and have them outnumber the ones with active receptors for the virus. Therefore, after reading up on this subject, it does seem as though gene therapy has promise but I do agree with a couple of the people who posted before me in saying that this is all relatively new and must be looked after very closely and tested in more people. But so far, so good.

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  13. The idea of gene therapy to control and treat HIV is very promising, especially since from what I knew, there are not many options for treatment for HIV. However, in reading this article, there were only 11 participants in the study, and some of them were not even able to be included in the results. The sample size and results of the study are not enough for me to be able to completely support the use of gene therapy, as there are probably some dangers that come with it also. I think before gene therapy can be completely credible for HIV treatment, more research needs to be done, with more study participants. However, I will agree that since positive results came from the study in the article, gene therapy is promising, but I think more research is needed.

    I am still a little confused about the process of this gene therapy, with the chemokines that we discussed in class though.

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    1. From not knowing anything about gene therapy until now I have to say I agree with your response and feel that gene therapy is a promising technique that will be very important in the near future to control the HIV/AIDS viruses. Although there is weaknesses with the results of the participants in this study (not all of them being included in the results) there is also positives. I as well can't support the study until they conduct it with larger sample sizes and also make sure all participants are accounted for so we can see if some of these promising results are in fact true! Having this incurable disease seems like there is no promising treatments, but after this study I believe there is so much hope for people suffering from this disease. It seems like a very good starting point and I'm very interested in learning more about this specific topic.

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  14. Ever since my first molecular biology class, I've always found gene therapy to be an interesting way to target various genetic diseases. I see it as an innovative and resourceful way to solve a lot of genetic health problems. We are using the body's own material to "rewrite" our DNA and our bodies, making them healthier. I believe that gene therapy has a promising future as a treatment for HIV/AIDS, and this study helped make that clear. Strengths were the positive results that they found in some of the participants, lowering the viral load to an undetectable amount is a strong feat. However, the study was weak in it's small n. There were only 11 participants, and some of them were not included int eh final analysis. This sample size is very small to support the generalizations made. However, this study does serve as a great starting point for more research involving gene therapy.

    Gene therapy is an innovative way to combat genetic diseases and should be further explored. It's also a great way to target HIV/AIDs from a public health aspect, working at a primary prevention level.

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  15. Haniya Saleem SyedaFebruary 5, 2014 at 3:16 PM

    This therapy look very promising and makes me fairly optimistic about the future of HIV treatments. However, I am still skeptical about how effective this therapy will be in treating the entire HIV population. First of all, the study only included 11 participants, 3 of which were not included in the study because of the low levels of the virus that latches to CCR5 and CXCR4. Another was not far enough in the study to be evaluated. This makes me questions the effectiveness of the therapy and the accuracy of the data presented. This information also makes me question how many people with HIV can actually use the treatment. If these 3 people did not qualify for the treatment, what does this mean for the HIV infected population? How many will actually be able to use this treatment? The sample size just seems too small to provide generalizable data.

    Secondly, the study says only 3 of the 7 participants achieved an undetectable viral load and only 1 maintained the undetectable status through week 19. Though this is promising, it still makes me question just how effective this will be.

    Finally, I understand this is just a brief article but there isn't enough information for me to really feel completely optimistic. For example, there is not much of a timeline presented. As far as we know, these results are only based on 19 weeks of therapy. It doesn't discuss any other possible effects or any other details about the participants, like their different viral loads. I'm curious to see how the therapy progresses, but this is still a great starting point.

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  16. I found the article “Gene Therapy Shows Promise in Controlling HIV” extremely interesting. I never knew that it was possible to control HIV through gene therapy; I previously thought that it was only antiretroviral treatment that was effective. One question that I have about gene therapy is about side effects. Antiretroviral treatment can have severe side effects, and each drug class has different side effects (Reust, 2011). For example, non-nucleoside reverse transcriptase inhibitors are associated with neuropsychiatric symptoms, rash, and liver toxicity, and nucleoside/nucleotide reverse transcriptase inhibitors are associated with lactic acidosis, lipodystrophy, and hyperlipidemia (Reust, 2011). Reading the gene therapy article made me wonder what kinds of side effects gene therapy for HIV treatment has on the body.

    Source:
    http://www.aafp.org/afp/2011/0615/p1443.html

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  17. I think gene therapy has a promising future for HIV/AIDS treatment. I’m glad to see progress in treating HIV/AIDS however there’s a lot more work that needs to be done. Although this method does seem effective for 7/11 of the participants who qualified, there was a very small sample size; therefore more research would have to go into larger sample sizes. Also follow ups need to be recorded to see how effective this treatment is, as it may only last a week, a year, etc, and other possible side effects it may have. Although this treatment may be only temporary, I do find it promising and am hopeful that brings us one step closer to finding a cure for HIV/AIDS.

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  18. To be honest, I do not have an extensive knowledge on gene therapy but I am extremely pleased to hear that more HIV treatments are at least being explored further than ARV (anti-retroviral therapy). It's important in the world of science that we do not become complacent with research and continue to explore our interests. After reading the article, "Gene Therapy Show Promise in Controlling HIV", I do believe this area of treatment has the potential to be gold standard and successful process of eliminating HIV and reducing our chances infection. There is proof that the genetic therapy extracting the functional CCR-5 gene, knocking out the expressive parts, and re-infusing it to the body does effectively reduce the viral load that HIV infected patients experienced. The downside of the research is the small sample size of 11 individuals. I think this research should be spread to other knowledgable, powerful scientific organizations who can take the results and really run with them, testing HIV patients who are willing and want to find a cure. Also, a downside is that there are also other causes of HIV, both genetically and epigenetically, so it is only a very small step in eliminating the virus from infecting humans altogether. Danny Lim's point about follow ups is also very important; we must follow up the participants to test how long the results last, whether there are any specific factors that inhibit the therapy and so on. All in all, I am very pleased to hear there has been progress in treating HIV/AIDS and trying to keep our population healthy from a virus that is a very serious threat.

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  19. My knowledge of gene therapy is limited but based on what we learned in class and the news article, I think it has a promising future in treating HIV/AIDS. As the article stated, gene therapy can be used to target the CCR5 receptors that the virus latches onto. Three of the participants achieved undetectable viral load for 8 weeks. Although this is a small number, some positive results were achieved so I think gene therapy is worth exploring to see if it has more potential to treat the virus. Since HIV/AIDS affects a huge population of people worldwide, it is important to follow any leads in terms of treatment.

    However, there are cons to gene therapy. For example, in the article, gene therapy can only be used against the virus if it is CCR5 tropic. Moreover, only individuals born with one non-functional version of the two genes were used in this study so the results cannot be generalized to all HIV patients. In addition, it is also worth considering that gene therapy is a relatively new treatment so there aren't any research available on how safe the treatment is. Also, I don't believe that any long term results have been achieved by gene therapy so a lot more research will have to be put into it. Finally, there are ethical issues regarding gene therapy. Some people believe it is intrusive because information about their genetic make-up can negatively impact their lives.

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  20. Like many of the other commenters have stated, gene therapy has always been really interesting to me, though I didn't understand much about it. The article "Gene Therapy Shows Promise in Controlling HIV", as well as what we have been going over in class this week made the subject a little clearer for me. I understand that HIV is transmitted by the virus binding our CCR5 chemokine receptors, and that in the kind of therapy being proposed, this would not happen because of of a therapy that knocks out genes that express the receptor. I think this a great thing, as it has proven to slow the progression of HIV, which is a disease that has so confused and flustered scientists and physicians alike for so many years. I wonder if this gene therapy can also work in a preventative function: such that individuals who do not have HIV can undergo genetic testing to see if they have the non-functioning CCR5-expressing gene. Then, individuals who do have this and feel like they are at risk for developing HIV due to other lifestyle choices can perhaps choose to undergo this gene therapy as a type of vaccine against developing HIV that is CCR5 tropic. This would definitely be an advantage to the use of gene therapy. However, a disadvantage to this still exists because even if this was developed, the cells of these people could still be infected HIV through CXCR4 receptors, as the article states. Could this gene therapy be done for the latter type of receptor as well, or are there too many genes which control the expression? Still, HIV is not caused only by the infusion of the virus into cells through these receptors- the virus has to enter the human body through contact with bodily fluids, and this still happens often, as 3 million people worldwide live for the virus. For this reason, in order to really stop the virus there is need for a cure, as these types of therapies can only slow profession, and research is still not clear on how long these therapies work for in the human body.

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  21. After reading the article I believe gene therapy does have a promising future as a treatment for HIV/AIDS. The Phase II clinical trial has shown positive results in one patient and potential in at least two others. Since scientists have yet to yield results such as these, any advancement is a good start. However, a few aspects of the study could be improved for further research into this method. Although this study is only in the Phase II trial, the sample size for this test was extremely small. Since researches have seen some success already, I think it would be best to increase the sample size tremendously. Additionally, the scientists are limited to those carriers with the "CCR5 tropic" and those who only have 'one non-functional version of the gene.' As with any new treatment study there are certain limitations. At this point, I think scientists should expand their sample size and if possible attempt to create a modified, similar treatment to work with subjects in which the virus also binds to CXCR4 receptors. Regardless of these limitations, the progress thus far seems promising. Although the idea of gene therapy may raise ethical issues for some, with control and proper education I believe people will learn to accept gene therapy in regards to life-threatening issues such as HIV/AIDS.

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  22. I briefly researched the CXCR4 receptor and found that HIV strains that infect via the CXCR4 receptor are only discernible late in infection. Based on this information, I understand why the researchers could not include 3 of the 11 participants in the final evaluation of the treatment. These participants were infected by variants of HIV that cannot be detected right away. I would like to know if these hard-to-detect strains, strains that infect through the CXCR4 are common. It seems that variants attacking through the CCR-5 receptor are more common, but I imagine there is still a portion of the HIV-infected population affected by strains that operates through the CXCR4 receptor. This treatment won't be able to control their HIV at all. It might even lead to disappointment in those who initially qualify for the treatment, but then find out they are ineligible due to having a strain that is not CCR-5 tropic. However, this study does exhibit gene therapy's promising future in HIV/AIDS treatment. It can only improve, and I hope that a similar therapy will soon be available to control CXCR4-operating strains of HIV.

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  23. I have never heard much about gene therapy before this article. However after reading this article, I can see that scientists are gaining more understanding about our genes. I find it very interesting that scientists are able to determine the connection between HIV with CCR5 receptors. The only criticism I had was that the study had 11 subjects, yet 4 of them could not participate. I feel as though they should have tested on more subjects to get a good sample size. Though their conclusion was that the infusion resulted in a suppression on the virus, I would like for them to test on more people to determine the success rate of their findings. Although I am still skeptical about gene therapy, I think it is a good start for controlling diseases. The scientists have already discovered the CCR5 receptor that HIV latches on, which shows the improvement and advancement of our knowledge and technologies. HIV is life-threatening and any help to control this can save a person's life. Once scientists are able to build upon this knowledge, we can possible control all diseases in the future. I am wondering if there are any cons to gene therapy?

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  24. Based on this week’s discussion on HIV, genetics, and the article, I think it is safe to say there is promise in gene therapy as a treatment for HIV/AIDS. Current ARV therapies are a lifelong process, and without them people with HIV/AIDS will begin to see a decrease in their health within a short time. Although I had to reread and go through the steps a few times to understand the full process, it seems as though these gene therapies have the potential to eliminate the lifelong treatments that many people living with HIV/AIDS have to deal with. Essentially, the process we talked about in class refers to the CCR5 receptor, which is where the virus initially enters our cells. However, those with the CCR5-∆32 mutation protect them against certain strains of HIV. If we can remove 32 base pairs and give people this mutation, the virus won’t be able to enter and replicate in the cells, thus protecting people from it.
    The strengths of this therapy are that, as that article mentions, there were long-term increases of CD4 cells in those who were treated. A downside of the ARV treatments is the continuous lifelong treatment. However, if gene therapy was done in a relative short term span, this eliminates the long-term aspect and provides a shorter treatment which improves the quality of life for those living with HIV. In addition, it significantly lowered the impact of the virus. Many people mentioned the small sample size number. I can’t argue that this is very true, and results certainly can’t be generalized to the general population yet. However, to support this research, I found an article from http://www.nature.com/nbt/journal/v25/n12/full/nbt1367.html that highlights a few other ways of doing gene therapy on HIV patients. It mentions altering protein-based inhibitors, RNA based inhibitors, T-cells, and others. As a few people mentioned, there are many steps involved in the process of the virus entering and replicating the cells. Even if using the CCR5 mutation helps currently, the virus may be able to adapt and form a way to replicate in a different way. However, research has clearly been under way using a variety of gene therapies that could prevent the virus from replicating in various ways. Therefore, I believe that gene therapy has a strong future in improving the quality of life of those with HIV/AIDS and potentially helping preventing some from developing the disease, as well as lowering the impact of the virus in those who already have the disease.

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  25. Before reading this article, and talking about gene therapy as a treatment option in class this week, I did not know much about this area of research. I found the article to be very interesting, and thought that the results were fascinating and promising. However, as a few of my fellow students mentioned there are definitely some major caveats in the case of this particular study. For instance, the sample size of the study was extremely small and consequently limiting- especially since only 7 out of the 11 participants went through the study completely. For this reason I believe that the evidence supporting gene therapy as a viable treatment for HIV is a promising start (it's kind of amazing that this study marked the "first evidence that sustained functional control of HIV in the absence of ART is possible,” isn't it?). However, more investigation into this realm definitely needs to be conducted.

    If anyone is interested in reading more about gene therapy in the news, I found this article about testing gene therapy as a treatment for blindness:
    http://www.technologyreview.com/news/519426/gene-therapy-tested-as-a-way-to-stop-blindness/


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    1. That's a really interesting article! It seems really amazing that gene therapy is opening up new ways to treat disease which otherwise seemed untreatable. Like the HIV study, the study seems preliminary but I haven't found any information that shows that gene therapy is not promising. It simply needs more research. It makes you wonder if maybe they will be able to cure blindness altogether!!

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  26. After today's lecture and the article posted, I think that there is certainly room for gene therapy in the future treatment of HIV/AIDS. As some people have already mentioned in their posts, one negative about these treatments is that they are just that, treatments. The idea behinds genomics and genetic testing should be to give people critical health information before they are diagnosed with a disease or virus; a preventative measure rather than a diagnostic measure.
    One thing about this study that bothers me is the small sample size. Out of the 11 participants in the study, 3 were not included because their cases of HIV/AIDS latches on to both CCR5 and CXCR4 receptors. Another participant is not far enough along in the trial yet for their results to receive an evaluation. These exclusions shrunk an already small group of 11 people down by more than a third. The result seem promising, but there need to be more similar studies done before I would call this groundbreaking news.
    People have also brought up the topic of possible side effects from the SB-728-T testing, and this is another problem that needs to be addressed. While the good news is that a company has found a potential treatment that will be extremely beneficial, this needs to be weighed against the possible, if not probable, side effects of a new drug. This SB-728-T might help a small percentage of the people needing it, but if it does more harm than good, is it really worth prescribing it to people? There is a quality of life that needs to be considered.

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  27. Based on this article, I do not think that gene therapy has a promising future as a treatment of HIV/AIDS. First of all, it says that these new cells could “control HIV for a sustained period of time without the need for ongoing antiretroviral (ARV) therapy”. Based on what we learned in class, out of the three different types of gene therapy it seems to be the kind that introduces a new gene (the engineered immune cells). Something the article does not state is how long these cells can control the HIV, and I wonder if it is for a significant amount of time, and how sustainable this treatment option is.

    Also, I am skeptical of the validity of this study due to the fact that it only had 11 participants, three of which were not included in the final evaluation of this study. Since the study included only 8 people at the end, I wonder if this treatment option is generalizable to the HIV positive population. The most positive part of the study to me was that this specific treatment caused an increase in CD$ cells in patients that was long term. In class we learned that this important in slowing down the diseases classification/progression to AIDS from HIV.

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  28. While I find the recent advances in gene therapy to be exciting and potentially promising, I can’t help but wonder how new treatments and tests will be paid for. The article “Gene Therapy Lurches Ahead, Sees Thorny Future Questions on Price,” discusses this issue. Treatments are not cheap, and insurance companies will likely do whatever they can to avoid being forced to pay for such therapy.

    This is similar to an issue mentioned in the article “My Medical Choice,” by Angelina Jolie. She states that gene testing and potentially lifesaving preventative treatment should be accessible to all women, regardless of their background or where they live. She explains that testing for BRCA1/2 costs upwards of $3,000, which is more than many women can afford. What Jolie fails to recognize is that if a person has trouble paying $3,000 for the testing, how are they going to finance surgery if the testing reveals they do have the BRCA1/2 gene and a mastectomy, for example, is recommended? While most health insurance policies in the United States (given the recent implementation of the Affordable Care Act), will pay for a percentage of the surgery, there are still high out-of-pocket costs that the patient will have to pay. Furthermore, $3,000 worth of medical care or testing may be better spent on cheaper interventions, such as immunizations, which are not available in all parts of the world. While we may have figured out how to test and treat genetic diseases using gene therapy, we have a long way to go in figuring out how to pay for it.

    Source:
    http://www.xconomy.com/national/2014/01/27/gene-therapy-lurches-ahead-sees-thorny-future-questions-on-price/?single_page=true

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  29. I think gene therapy has a promising future for HIV treatment. The treatment lead to suppression of viral load in the blood which is what we learned in class would slow down the progression of HIV to AIDS. I read another article related to this one. It discussed the pros and cons of gene therapy. One of the pros was using gene therapy to cure genetic disorders because this is the only way it can be treated. Another benefit is to target the reproductive cell of the genetic disorder and stop it from being transferred to future generations. Gene therapy can also have many advantages over drug therapy. A few of the negative effects mentioned include lack of knowledge, need for long-term results, not as safe as drug therapy, and risk of causing more damage. It was definitely an interesting article and it went on to discuss ethical issues as well. I recommend it to anyone interested in the subject.

    http://www.gtherapy.co.uk/pros-and-cons

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  30. http://www.ted.com/talks/seth_berkley_hiv_and_flu_the_vaccine_strategy.html

    In class we learned how HIV infects and replicates in the body but I was curious to know how the body normally responded. I found a Ted Talk with a great animation (starting at 4:50) that shows us how the body would respond to an HIV infection if it was vaccinated prior to infection. Epidemiologist Seth Berkley says the body would normally do this process in a much longer time period (about 7 days) and by then the battle would be lost. Starting at about 9:55 he begins to talk about creating a vaccine from broadly neutralizing antibodies that latch onto the virus (no matter how frequently it changes). This utilizes binding sites on the virus that are similar to how the virus enters a somatic cell.

    In regards to the new treatment models we learned about, I believe the pros are that they offer a much longer life expectancy for people infected with AIDS. They also allow us to learn more about the virus and improve our treatments further. However, I believe a major con is that this is focusing on treating the virus when a more beneficial approach would be to prevent it altogether. This ted talk offers a suggestion for this. I found this shift from treatment to prevention (a common theme in public health) an interesting addition to class material.

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  31. I definitely think that gene therapy has a promising future as a treatment for HIV/AIDS. Being able to block the CCR5 receptors on the surface of the CD4 cells to stop the multiplication of the virus DNA is a positive step forward in treating HIV/AIDS. I find this new proposition truly fascinating and after some further research it seems this area is not only promising for treating HIV but also many other diseases.
    At first I was confused to how gene therapy would actually treat HIV. It seemed from our discussions in class that gene therapy was just another way to manage HIV rather than taking drugs. The article further states that gene therapy can be a delaying mechanism rather than an actual treatment. Still the indication that the treatment led to long term increase of CD4 cells and a drop in the viral reservoir shows that with further treatment that the virus could potentially be reduced and even eliminated. Furthermore, as we discussed in class today, HIV relies on continual replication of itself, so if we can stop the replication perhaps HIV could be eliminated.
    Molly stated above, a problem with gene therapy and this study is a lack of research. There were only 11 participants in this study and only 7 of them actual produced sufficient data. Clearly more research is needed in the area to make gene therapy reliable and safe. With limited studies being done we don’t know what the effects could be with gene therapy. Perhaps manipulating genes could cause some adverse effect for the patient, we don’t know! Maybe gene therapy will move from being a treatment to a preventative measure to stop HIV infections all together. This idea brings up a whole new set of ethical issues. Yet still, I am excited about the promising future that gene therapy holds. Anything that has positive signs of improving health is at least worth further research.

    This short article about gene therapy treatment of a skin condition shows how gene therapy can have long term health benefits. The article reports back on a patient who received treatment 7 years ago and his condition has drastically improved. Although this is not about HIV i think it is important so show that gene therapy is growing in success and we should be optimistic about the future use.
    http://www.sciencedaily.com/releases/2013/12/131226143110.htm

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  32. I, like many of my classmates, think that gene therapy is a promising technique. While this article supports this claim, it does have some weaknesses. For example, the fact that there were only 11 test participants combined with the fact that not all of them were included in the evaluation, means that these results may not be generalizable to the general public. However, some of the participants did have some promising results, which it would be interesting to see if they could be replicated in a study with a larger sample size. I also wonder if there would be similar results if some of the methods of gene therapies that were discussed in class were used.

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  33. The article is interesting, but I don't think that gene replacement is a viable treatment method. It can be a "working-cure" which is excellent for HIV patients (who can afford what I can only assume is a fairly expensive and long-term procedure) but leaves reservoirs of the virus in the body. From the public health perspective, it is more interesting to look at the potential of gene replacement therapy being used in the same way a vaccine is used, for high-risk individuals, but obviously only possible if the technology surrounding GRT improves.

    This article (http://news.sciencemag.org/health/2010/06/can-gene-therapy-cure-aids) goes into a case study in more detail about techniques being used to attempt to cure HIV using GRT, if anyone is interesting. Personally I agree with it, in that GRT as of now is only a replacement for antiviral drugs in that it can lessen the side-effects of HIV but not cure the disease itself.

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  34. As many students have discussed, despite the flaws with this study (11 participants, 7 evaluated, only 3 achieved undetectable viral load) the results are very encouraging. I think gene therapy is an auspicious technique as treatment for HIV/AIDS as well as many other diseases. There are many benefits to gene therapy, such as limited side effects compared to drug cocktails, no drug resistance or tolerance, and most importantly, providing a cure instead of delaying the disease or easing the symptoms. This study reminds me of the treatment done on Timothy Ray Brown, "The Berlin Patient" in 2007. He had leukemia and the doctors did a bone marrow transplant from a "donor with a rare genetic mutation called CCR5 delta32 that makes stem cells naturally resistant to HIV infection" - the same gene used here in gene therapy, he is thought to be the first man "cured" of HIV and he still has undetectable levels of HIV. Unfortunately, the same treatment was tried on two Boston patients, and it was thought to have worked, but 32 weeks after treatment, HIV returned. These results demonstrate that we are on the right path, but there is still a lot of research to be done.
    Here's a link to the two Boston patients.
    http://www.cnn.com/2013/12/07/health/hiv-patients/

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  35. Based on the evidence provided, and on emerging mechanisms of gene therapy, I think there is a promising future for the development of a treatment for HIV. Although some of the patients maintained an undetectable viral load for an extended period of time, it was only effective with 3 out of 7 of the observable participants. Clearly this particular treatment would require a much higher success rate (and a larger scale of study participants) before it could be considered "successful", but it does encourage and support further investigation of the control of the CCR5 receptor via genetic therapy.

    This treatment is promising for the future of HIV treatment, but it is not a viable treatment method. That being said I excitedly await new approaches for treating HIV through gene therapy.

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  36. The amount of the subjects tested and evaluated is too small. I would like to see this study done with a larger sample size such as 100 people with HIV. However, I do think gene therapy has a promising future because it 'customizes' treatment for individuals. Gene therapy can help eliminate the trials and errors of testing several medications. With gene therapy, providers and scientists can narrow treatment by understanding the specific genes that are affected. The one problem I see with gene therapy is the cost. I feel like gene therapy would be expensive because it is advanced.

    In class I learned that HIV can get into a host T-cell by binding to CCR5 receptors. I also learned that deletion in the gene 32 is a protective factor because it prevents HIV from binding to CCR5. I though this was interesting because whenever someone mentions mutation or deletion in the gene, I automatically assume a horrible adverse effect however in this case it is beneficial.

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  37. I think gene therapy will have a promising future as a treatment for HIV/AIDS. Coming this far with being even able to do with a huge epidemic that occurred long time ago and coming to a point being able to put a stop to it or prevent it, it has been a great achievement. Gene therapy is basically manipulating the blood cells and basically picking out the bad and putting in the new genetically engineered cells into the body. The thing now it has limitations only due to the fact is only works for the CCR5 cells compared to the other cells that also need to be targeted. The strengths are that it has been working for many other people in the experiment but it did work for many other people that have been participated. I feel with how much they have achieved to this point, they have the ability to target all the mutated cells having to do with HIV/AIDS.

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  38. After reading the article "Gene Therapy Shows Promise in Controlling HIV," I have come to understand a little more about the future benefits of gene therapy in the treatment of HIV. As we learned in class, HIV binds to the CCR5 receptors in a host T-cell as its initial method of infection. The article discusses the knocking out of genes whose expression results in the activation of CCR5 receptors, which would prohibit the "tropic CCR5" HIV virus from latching onto T-cells. Though results do show promise, I do hold one hesitation in that the sample size of the study was very small. I would hope, in the future, that a larger sample size would be used to get a better gage of the effectiveness of this new therapy on a population with the topical CCR5 HIV virus.

    To better understand the implications that must be considered when developing new gene therapies, I found the article "Partial Considerations in Gene Therapy for HIV Cure," to provide helpful insight. Page 4 of the article lists the following obstacles that prevent the application of gene therapy to cure HIV infection: (a) the difficulty of applying a rare disease therapy, such as gene transfer, to the large AIDS population; (b) resolving the
    safety of cytotoxic conditioning required for cell-based gene therapy; (c) clinical trial design issues, including: 1, correct identification of target population based on thorough risk-benefit analysis, and 2, the lack of validated cell-based endpoints defining efficacy; (d) development of a safe method for selection of gene-modified cells; and (e) the restrictive cost of manufacturing and technology. What I found most helpful in relation to the assigned article was part c, which discussed clinical trial development. As stated previously, one concern that I had with the previously mentioned study was that the sample size was small. After reading this second article, it makes sense that initially the sample size would be on the small side, as it could be difficult to find patients who are at the exact stage of the disease and/or fall into the target population category.

    Though at first I struggled with understanding the basic components of HIV infection on a genetic level, it has become more clear to me when reading about gene therapy studies and how researchers have thought about HIV infection in reverse in order to discover beneficial treatments.

    Source: http://download.springer.com/static/pdf/10/art%253A10.1007%252Fs11904-013-0197-1.pdf?auth66=1391908735_c711aac4b5c13dd90f54e6930cca355b&ext=.pdf

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  39. This gene therapy article is quite exciting because, as it states, "this is the first evidence that sustained functional control of HIV in the absence of ART is possible." ART, while successful in many cases, is very problematic in others. They require that patients strictly adhere to complicated medication regimens, they are very costly, and they often come with many side effects, with some being more severe than others. An alternative treatment option to ARV could revolutionary in controlling HIV. However, I'd imagine that once this gene therapy technique became a viable option, through more trials, it would be even more expensive than ARV. However, at least with this treatment option, it only requires a single infusion, as opposed to long term ARV treatment. Hopefully, over time, the price could be reduced to something more affordable. Nonetheless, much more research would need to be done in gene therapy before it became a treatment option. This study only had 11 participants at the beginning. This already low sample size was then reduced to 7. While this data alone isn't usable, it does point to a promising future. It will be exciting to see where this gene therapy research leads.

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  40. I agree with most classmates in that gene therapy for HIV is promising although it is not proactive enough for my taste. The sample size is tiny, but promising. However, public health concerns much broader populations and whether people like it or dislike it, funding and distribution is a huge factor. Gene therapy is extremely costly and will have to be put through rigorous testing which includes much more people and their results. Being proactive means we must define gene therapy as a viable way to eliminate HIV altogether in patients as this is the only way benefits would outweigh costs. For most people gene therapy is a far too expensive luxury and to add another concern it is very unpredictable. Without knowing what future risk it poses to patients (such as creating a more violent strain of disease or having adverse effects on genes in the body) we will not know if it is the best option for widespread cure. This being said I think gene therapy can help scientists learn more about the specific genes that contribute to the overall puzzle, thus helping discover a more effective inexpensive treatment. Currently gene therapy stands on the fence...a pandoras box or a miraculous cure all, either way the process needs to be perfected and then we can start discussing if we think it is a watershed component in public health for the fight against HIV, for now it is only speculation and predicted potential.

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  41. Gene therapy wasn't the first thing that came to my mind when thinking of treatments for HIV, but as many others said, it is method with a promising future. The results of the study discussed in the article provided very positive and promising results, however the main critique that I have, which has been a popular one, is the small sample population that the study used. Considering that only 7 of the 11 total participants actually partook in the study doesn't say much for the total population of individuals who are at risk or are currently dealing with HIV. I do think that in order to further the development of gene therapy as a treatment for HIV, more studies with larger sample populations need to be conducted to make sure it is as efficient and beneficial to the greatest number of patients possible. As a whole, gene therapy provides a great amount of hope and positivity to working towards a cure and to help eliminate the virus. Further implementation is definitely essential to the overall success of gene therapy as a HIV treatment. I hope there is more work done with gene therapy and HIV in the future!

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  42. I found this news very interesting and exciting. The fact that patients suffering from HIV will be able to better control their disease without the use of ART provides so much hope for the millions suffering.

    I am a little confused however as to the logistics of the studies being conducted. How are these studies being funded, and will funding continue to be provided? Also, how would the results differ if the studies had been conducted with a larger study population? In addition, if such a large proportion of the participants were not qualified for the study, how would this affect the availability of treatment to HIV patients in the future?

    Although the results of the study were very promising, these points do make me question the validity of the study.

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  43. I think gene therapy does have a promising future in controlling HIV. I think a major strength of using gene therapy is that it can be personalized - your set of genes can be manipulated to control contracted HIV. This specific gene therapy in the article targeted CCR5 receptor genes and manipulated them to inhibit entry of the HIV virus. The new therapy only worked for 3 of 11 study participants, however this is because we all have a different genetic makeup. In the future, scientists can work on therapies that will address the many different ways the HIV virus enters a host T cell, not just the CCR5. For instance this study highlighted that the gene therapy they were testing didn't work on three participants who had virus that also latched on both to the CCR5 and CXCR4 receptor. Hopefully a future therapy can address this situation as well. It would be amazing to one day be able to draw blood from an HIV positive patient and be able to tailor a gene therapy to an individual's need.

    A limitation to gene therapy is that it is a new field and therefore there is a lot of uncertainty, risks and possible unforeseen long-term side-effects. We must take caution and really know and understand the risks before gene therapy can be widely used. Also a limitation of gene therapy for HIV is that although it shows to be an effective treatment for controlling HIV in this study, it is no cure.

    Overall, gene therapy seems to be an effective treatment that could greatly bring down the incidence of AIDS in the future, however it is still a new field and requires much more investigation.

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  44. I definitely thing gene therapy is the way to go for future HIV treatment, and can probably be applied to cancer treatments or other diseases where there is a genetic factor. I am, however, skeptical of the study due to the low amount of patients enrolled. I do think it is a promising start and would love to see it studied in a larger population and see if the results hold.
    I do have a question about gene therapy though. How exactly is the new gene incorporated into a person? Is it through medication or some other sort of medical procedure? I know the article talked about re-engineering red blood cells but do they alter the receptors of every blood cell? I would like clarification.

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  45. When I read this article, I did not know exactly what antiretroviral therapy (ART) was; after doing an online search, I found that the standard ART “consists of the combination of at least three antiretroviral (ARV) drugs to maximally suppress the HIV virus and stop the progression of HIV disease.” http://www.who.int/hiv/topics/treatment/art/en/index.html
    In addition, I also found that ART could have adverse effects such as increasing the risk of developing cardiovascular disease, diabetes, and osteoporosis. http://aidsinfo.nih.gov/guidelines/html/1/adult-and-adolescent-arv-guidelines/31/adverse-effects-of-arv

    From what we learnt in class about gene therapy, I think it may be the closest “cure” we potentially have for HIV without the need for ongoing ART. Ideally, these engineered immune cells should function as normal CD4 cells, but without the presence of CCR5 receptors. The CCR5 receptors are the beginning of HIV infection, and to eliminate it is to prevent the whole process of infection. If scientists perfected this kind of gene therapy for all HIV-infected persons, it would be beneficial for everyone in the long run; patients won’t have to endure multiple visits to the doctors and health care costs will decrease. However, much precaution must be taken before finalizing gene therapy for HIV patients. Like ART, there can be adverse effects to it like our lymph system overreacting to these engineered cells. Moreover, more research should be done on HIV gene therapy until it can be declared as a legitimate intervention for HIV. But so far, the future for HIV patients seem promising.

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  46. I think gene therapy offers a promising future for HIV treatment. Current HIV treatment requires a daily cocktail of antiretroviral medications over a lifetime, referred to as antiretroviral (ARV) therapy. While ARV therapy is effective at reducing the effects of the HIV virus and reducing HIV transmission at the population level, it is tedious and costly for patient. Patients on ARV treatment must be disciplined and take their medications regularly. This can be extremely difficult to track. Furthermore, ARV medications are expensive and can cost HIV patients a fortune over a lifetime. Gene therapy would offer patients the ability to obtain treatment at a one-time cost with no need for a cocktail ARV treatment regimen.

    The potential to offer gene therapy for HIV treatment is extremely beneficial from an efficiency and quality of care standpoint. However, the obvious limitation with this treatment is that has not been tested rigorously. This study alone only had 11 participants, 7 of which were found to be eligible for the gene therapy treatment. More research on gene therapy must be done with larger sample sizes in order to confirm its effectiveness and feasibility.

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  47. I think the use of gene therapy to treat HIV is really incredible. Based on the article above, it seems that there is promise for gene therapy. However, I think it is important that they expand study on it and it's effectiveness and benefits. Which leads me to a question: what are the benefits/strengths of gene therapy and do they trump the benefits and use of antiretroviral drugs?

    In contrast to what we have been discussing in class, how we can treat HIV and what interventions are possible ... I wanted to share this video where the HIV virus is actually helping someone with her morbidity. This video features a young girl with leukemia, and the use of the HIV virus to kill her cancer cells.

    http://www.ryot.org/video-hiv-used-cure-dying-6-year-old-cancer-patient/482505

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  48. The path that gene therapy is paving is truly amazing. Who knew an idea conceived several decades ago would become a running alternative method to treating genetic diseases. After reading "Gene Therapy Shows Promise in Controlling HIV," I have no doubt that this alternative method of treatment will be successful, but not for another several years. Just like those above me have stated, the study is unreliable at the moment. With the study having only 11 participants, the sample is really small, no way close to even representing a population. Once these scientists have extended their research to include hundreds (and even a thousand) participants will only then the study will be reliable.

    For now, this study can only raise hope for those affected by HIV. In the meantime, the best bet is to continue using available treatments and medications already available for patients with HIV.

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  49. It seems to me that the incorporation of gene therapy as a form of treatment for HIV/AIDS and other life threatening diseases can be very beneficial to society as a whole. I found an article about the use of gene therapy in children that are born with a disease in which they lack immune defenses. This immunodeficiency disease is called SCID-X1, and the gene therapy includes using a “self-inactivating” virus to deliver and introduce the correct gene into the children’s blood stream. The introduction of these new cells decreases the risk of these children succumbing to their deficient immune systems. They are also showing no signs of cancer, which was a side affect in children with this disease that were given earlier versions of this gene therapy about a decade ago. With the success of gene therapy with SCID-X1, researchers in Boston are now looking to create similar treatments for sickle cell disease and thalassemia. It is exciting to see that further research is being done in order to develop and improve new gene therapy treatments for diseases that affect many people in the US and around the world.

    Source: http://www.npr.org/blogs/health/2013/12/07/249335960/gene-therapy-keeps-bubble-boy-disease-at-bay-in-8-children

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  50. I definitely agree that gene therapy is a promising medical breakthrough that can change the way we treat HIV. However, I do agree with other students that this study has yet to show reliable findings as there were only 11 participants. Another limitation that I found is that the treatment only works for viruses that use CCR5 receptors. What about those other viruses that do not use the CCR5 receptors? This study may have positive results, but that's mostly for those who have viruses that are CCR5 tropic.
    Although many questions are still being raised, I can see this new gene therapy opening doors to many other innovative researches regarding the control of HIV. Moreover, these findings can lower the cost of treatments for people who are still relying on continuous ARV therapy. HIV treatment still has a long way to go, but these researches are contributing enormously to the list of methods that will hopefully be proven to be successful someday.

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  51. As I read through the article “Gene Therapy Shows Promise in Controlling HIV”, I cannot help but feel excited and optimistic that science is heading down the right path of finding a solution for HIV. HIV is a disease that many people suffer from and most often times cannot get rid of. Though this new research regarding the control of immune cells for HIV is not perfect, it’s on its way to saving many lives and shows signs of hope. People are not expected to take the back seat and live with HIV, if scientist can use gene therapy to even help a small amount of the population, then this is a step in the right direction. Scientist will continue to take the information from studies and from these eleven participants and tweek how they approach the study. HIV is a deadly disease that effects children and adults all over the world, finding a cure for HIV without relying on treatment will not only be a scientific breakthrough for HIV, but for other incurable diseases as well.

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  52. After reading and talking about gene therapy in class, I think it has great potential to treat diseases previously considered incurable. Medicine is an ever-changing field, as new research and practices are introduced regularly. Gene therapy, while still a relatively new treatment, has proven to have very positive outcomes. Diseases like cancer and HIV are extremely scary to patients, because they are dangerous. In an article in The Economist this week, gene therapy researched is discussed. The article talks about the research of Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy, who uses a cell vector derived from HIV to insert the correct gene into the target cells. This is interesting because we are conditioned to think of the HIV disease as dangerous and deadly, but the way that it infects a cell could be extremely helpful in healing broken genes.
    The Economist article:
    http://www.economist.com/news/science-and-technology/21595888-fixing-bodys-broken-genes-becoming-possible-ingenious

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  53. Reading the article "Gene Therapy Shows Promise in Controlling HIV" made me very hopeful for possible cures to diseases such as HIV. At the same time, I was aware of how many small success stories there are of gene therapies that never make it to the public for clinical use. This article did make the treatment sound promising, since there was a significant improvement in the patients even without the use of antiretroviral therapy. The clinical trial should be repeated so then it can be applied to the public on a much broader scale. I found an interesting article from The Economist about gene therapy on several diseases other than HIV. This includes liver disease, Choroideremia, metachromatic leukodystrophy, Wiskott-Aldrich syndrome, and more. It is interesting to read not only about gene therapy on HIV, but also these other illnesses related to genes. The link to the article is posted below.

    http://www.economist.com/news/science-and-technology/21595888-fixing-bodys-broken-genes-becoming-possible-ingenious

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  54. As many of my classmates have mentioned, this study holds incredible potential for the future of HIV/AIDS treatment. Despite the limitations of this study, where only 11 people participated and 7 fully completed it, the results are promising enough that future trials should be conducted. I honestly hadn't considered gene therapy as a viable treatment prior to this class; it had seemed like a treatment in the realm of science fiction, or a distant possibility that wouldn't come to fruition anytime soon.

    Like Sarah and Michelle, I'm concerned about the funding of these therapies. Although gene therapy may become commonplace in the future, it is incredibly costly now. If this were to be a viable treatment option for HIV patients, how could they begin to afford this? What options would they have, considering that most insurance companies wouldn't cover this? Michelle linked a fascinating article (located at http://www.xconomy.com/national/2014/01/27/gene-therapy-lurches-ahead-sees-thorny-future-questions-on-price/?single_page=true) that covered this exact issue. In gene therapy, there's only one injection, which could cost more than thousands of dollars.

    As another article pointed out when the Children's Hospital of Philadelphia funded a gene-therapy company, the expense is high - and not everyone may be able to afford it.

    (source: http://articles.philly.com/2013-10-24/news/43327514_1_gene-therapy-treatment-gene-therapy-gene-therapy-company)

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  55. After reading the article "Gene Therapy Shows Promise In Controlling HIV" I was surprised by the innovated treatment for HIV. However, I feel that the success of the experiment was assumed a little to early considering the number of participants and the short amount of time that the experiment had been conducted. I don't mean to discredit the results that have been found, I more mean to say that we should wait to announce the results until more data has been collect. 11 participants is an extremely small number to base an entire population of HIV victims on, and only 7 of these participants had enough virus receptors for the experiment to work with the gene therapy. This is a huge step in the fight against HIV, but it still seems that more research needs to be done before researchers can fully prove the validity and consistency of their new findings.

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  56. Although I think gene therapy may be helpful in the treatment of disease in the future, the current techniques are risky and the associated benefits remain unknown. However, this is not to say that I am not hopeful advances in medicine, genomics, research, and technology may eventually lead to the success of gene therapy for diseases such as HIV. In the article Gene Therapy Shows Promise in Controlling HIV, I was intrigued by the method used by researchers to suppress the amount of viral DNA in the blood. Ultimately, what caught my attention was the small sample size. The data makes the case for gene therapy stronger, but I would like to see the results from a larger population as well as follow-up studies.

    I found a great article, published in October 2013 in the Genetic Engineering and Biotechnology News, HIV Researchers Seek a Potential Cure that references the above study by Sangamo BioSciences. It mentions that currently antiretroviral therapy (ART) is the most regarded type of treatment. However, this method does not restore normal CD4 cell counts in a majority of subjects. The article goes on to further explain other types of treatments such as Stem Cell Therapy as a potential cure for HIV. There seems to be a few types of treatment possibilities heavily invested upon in research and I am optimistic that one (or more) will prove to be effective in the near future.


    Article: http://www.genengnews.com/gen-articles/hiv-researchers-seek-a-potential-cure/4997/

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  57. Because this discovery is "the first evidence that sustained functional control of HIV in the absence of ART is possible,” which points to an important breakthrough with major potential upside, my opinion is that gene therapy does in fact have a promising future as a treatment for HIV/AIDS. As aforementioned by numerous classmates, there are certainly weaknesses that are hand-in-hand with this experiment, such as the minuscule number of study participants, which was limited to 11 and then even further bogged down by complications. Moreover, the scope of the study was only targeted towards a specific population, the HIV-positive with only one-of-two active receptors on the CD4 cell, so these results cannot be applied to all of the HIV-positive, never-mind the inherent inapplicability to the general population that is HIV-negative. Although the setbacks to this discovery are very real, the fact of the matter is that this breakthrough is a step in the right direction and was significant enough to be brought forth in the 53rd Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC), which bodes well for opening up more in-depth future research with more thorough experimentation and analysis. Gene therapy is here to fight HIV/AIDs, and is just getting started.

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  58. After reading this article and from what we learned in class, gene therapy seems like a good step in the right direction towards curing HIV. However, I agree with what Naziyya said above about how this study has some limitations. This study has limitations from the small sample size of 11 people and also only 7 were able to complete it. Therefore, I think it is necessary to conduct further research on this topic to really discover its reliability and validity.

    I agree with that fact that the price of gene therapy is a problem. In this article from the LA times (http://articles.latimes.com/1990-11-08/business/fi-5652_1_human-gene-therapy) it states that the new pricing paradigm of gene therapy must "strike a balance between pharmaceutical profit and public health" in order to be cost effective enough for people to go through with it. Despite the fact that it is pricy, my question is, if we invest in gene therapy in curing certain diseases, how much money would we save in other areas such as, medications, treatment, interventions, etc? That would be helpful to know when analyzing the costs and benefits of gene therapy.

    Aside from the price, I think gene therapy is a good step in the right direction to curing diseases that were otherwise known as "incurable."

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  59. After reading the article and learning that gene therapy for HIV attempts to change a heterozygous expression of CCR5 into a homozygous allele, I'm curious about the future impact of this gene therapy. If the homozygous allele is more favorable, as it provides almost complete protection against HIV-1, would that become a favorable adaptation through natural selection? Could at some point in the distance future, everyone be homozygous for the CDR5 allele and thus protected against HIV-1?

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  60. This article may not convince many people that gene therapy has a future in the fight against HIV/AIDS due to its various weaknesses and its poor results, but I do think gene therapy is the future of HIV treatment and prevention. AZT was an incredible breakthrough that has allowed millions of people with HIV to live prolonged, happy lives. I think gene therapy is the next step. There is clear evidence that geneticists have found a gene that in a certain state makes people almost completely immune to HIV. Why not put the necessary resources into more research of this gene that could potentially lead to a vaccine or help people already with the disease become cured? One argument I kept seeing against this was that immunity to HIV would encourage people to participate in more dangerous behavior, but I think this argument is premature and assumes the worst in people. CCR5 receptors and CD4 cells have clearly been shown to greatly contribute to HIV infection. We have a duty to pursue the greatest possible treatments for diseases, especially a potentially deadly disease like HIV/AIDs that affects so many different people. Gene therapy for HIV/AIDs is promising, but would most likely be very expensive. One could argue a gene therapy to treat AIDS would only be available to the most affluent. I view this issue from a utilitarian perspective in that the resources being poured into research will pay off and the benefits will highly outweigh the risks. I attached an article that many people have probably read or heard of that I find fascinating about a man who was potentially cured of HIV.

    http://www.foxnews.com/health/2012/09/13/berlin-man-doctor-convinced-hiv-cure-is-real/

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  61. Gene therapy definitely has a promising future in controlling HIV/AIDS, although I wouldn't necessarily say that it is promising as a treatment just yet. My main concern is that the therapy was only tested on eleven people and found successful in three cases. This seems like way too small a number needed to make a claim that gene therapy could be a successful treatment for HIV/AIDS.

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  62. After this week's discussion and reading the news article above, yes, it does seem that gene therapy is headed in a positive direction in terms of treating HIV. Like many of my colleagues, the sample size of the study is very concerning. With such a significant portion removed out of the original eleven participants for final analysis, and only less than 50% showing beneficial results of achieving undetectable viral load, the study does not present itself as a "game-changing" revelation.

    Nonetheless, the small sample of subjects is understandable as these are human subjects in a study involving modifying genes that regulate the immune system. Any unexpected or adverse effect can be detrimental. Thus, even three people showing such results should be regarded as progress in a still emerging field. It is known that a substantial portion of the natural population has mutated versions of the CCR5 receptors, and they do not present any immunodeficiency. Using that ideology, this study has the right intentions and basis for possibly bringing gene therapy as a viable means to HIV treatment. Perhaps the most astonishing result of the study is the long-term increased CD4 cell count. As these cells play a vital role in messaging immune cells to fight off pathogens and foreign bodies such as HIV, this is great news for gene therapy moving forward. The fact that this therapy is even being tested on human subjects right now shows that there has already been considerable progress at the animal testing phase. Therefore, a few adjustments to the current approaches in gene therapy can possibly culminate soon in the future, an effective treatment for HIV.

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  63. After reading the article, "Gene Therapy Shows Promise in Controlling HIV," I believe that gene therapy has a promising future as a successful treatment for HIV/AIDS. Even though the treatment was proven successful for only 7 patients out of the 11 participants, I still believe that this is a step in the right direction. If more research is conducted and a larger patient population is used for future studies, researchers will be able to prove the reliability of this treatment. I believe that a limitation for this study was in fact the sample size and I believe it is important to have a much larger sample size if you want to produce accurate and convincing results. Having a larger sample size will be a more accurate representation of the population you want to target with this gene therapy. I would be more convinced to use gene therapy as a treatment if it was proven successful for a larger sample size. However, the important thing is that this study did produce successful results, and I think this is crucial for proving that gene therapy can be a promising treatment for HIV/AIDS.

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  64. I think gene therapy could definitely have a future role in controlling HIV/AIDS. However, due to the small sample size, it's hard to tell from the information extracted from this study whether or not gene therapy will play a large role. I found it interesting how varied the results were, even with only eleven people. I didn't know that HIV can attach onto multiple receptors, so that changes my opinions slightly on how we can combat HIV/AIDS. Because HIV can attach to multiple receptors, the job of preventing/stopping HIV is much more difficult that if it were only one receptor. This means that any cure/treatment we find may not even work for half of all HIV/AIDS patients, since in this study only three of eleven cases were reported as subsided. My biggest take from this article and from this week is that ending HIV/AIDS will be more difficult than many people think it will be, but we can start by doing things like gene therapy.

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  65. The ongoing research and discoveries that have been made in the field of genetics is truly remarkable. Gene therapy in particular, has made great strides in medical advancements over the years; it has had promising results in both genetic disorders and acquired diseases. The idea of being able to introduce copies of healthy genes into people who lack them is fascinating and a step in the right direction towards potentially being able to cure deadly diseases such as HIV/AIDS. Using gene therapy to help treat HIV/AIDS, as mentioned in the article, is something that I have never heard of, however it seems like a promising way to perhaps being able to treat this illness. The article mentions that this new gene therapy led to a long term increase of CD4 cells and a corresponding drop in the virus leading to sustained functional control of HIV. This is something that we have to look into more; it is something that could prove to be extremely effective, with further research. However, as many mentioned, there are limitations to this study as shown by the limited number of participants and, more importantly, with the price factor. Gene therapy, in general, is quite expensive and without a strong case being made for it, in terms of successful follow-up studies with a larger sample size, it may not prove to be cost effective as only the affluent may be able to afford it.

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  66. I do think gene therapy has a promising future as a treatment methods for HIV/AIDS. Treating a disease that attacks on the micro level through micro alterations seems rather promising from a basic logical stand point. But, the fact that such a treatment has shown even a small level of success stands as even more promising proof that gene therapy could one day be the solution to the HIV/AIDS disease.

    Granted, as this article shows, it will take many small steps before we get to a point where we can apply the developed treatment to a large group of participants. We may also run into several more walls with regards to gene therapy as, just like each and every one of use, our genes too are different. We may run into the problems where one form of treatment works on some individuals and not others, as was the case in the article's study group.

    But, even a small bit of advancement now, can later lead to leaps of advancement.

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  67. The article "Gene Therapy Shows Promise" surprised me because this is a huge breakthrough for HIV patients. It is so exciting to a HIV patient to be able to go a certain amount of time without ARV treatment. I dont know how affective this treatment is because the sample size is really small.
    In another article by Willy Bogers, there has been a breakthrough for a vaccine that affects the CCR5 receptor in the vaginal area so HIV infection cannot infect through the vaginal rout. They have found that this vaccine inhibits one certain HIV infection from infecting someone. This also excites me because this can prevent some HIV that is transmitted through intercourse. Its so fascinating to me how much research is finding cures for certain diseases and how far we've come even in the past 10 years.

    Source: http://www.aidsmeds.com/articles/Sangamo_genetics_1667_24579.shtml

    http://journals.lww.com/aidsonline/Fulltext/2004/01020/A_novel_HIV_CCR5_receptor_vaccine_strategy_in_the.3.aspx

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  68. After reading "Gene Therapy Shows Promise In Controlling HIV," and analyzing some of the information that I learned in this week’s discussion on HIV, genetics, and treatment, I do in fact think gene therapy has a promising future as a treatment for HIV/AIDS. Having said I believe there is a promising future for this type of treatment, I think it will take a lot longer to produce consistent positive results, than some people expect. I believe it will take a lot longer because in the article "Gene Therapy Shows Promise In Controlling HIV,” the study sample of participants was very small (11), and on top of the small study size not every study subject could participate because they did not meet the requirements in some way, whether it be the case of HIV they had latched onto both the CCR5 and CXCR4 receptors, or if they were not far enough along in the trial yet to obtain an evaluation.
    Having said all of this, I still believe the treatment process will eventually provide positive results to large numbers of people because of the results that were displayed by Dr. Dale Ando in this study, although a small number of results were displayed, they show that the control of HIV is possible. In support of my last statement, I feel this quote by Dr. Ando exhibits my main reasoning for my opinions; “The data…demonstrate that a single infusion of SB-728-T can lead to profound suppression of viral load in the blood and sustained functional control of the virus,” Dale Ando, MD, Sangamo’s vice president of therapeutic development and chief medical officer, said in a release. “This is the first evidence that sustained functional control of HIV in the absence of ART is possible.”

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  69. This articles gives me mixed emotions. On the one hand I find it to be exciting and inspirational. It gives me hope that this could be a stepping stone for finding a new way to treat HIV without all of the awful side effects that ARTs are known for. It is also might provide a guide for other researchers to develop a gene therapy for the other strains of HIV that do not only use the CCR5 gene.

    However, I am a tad skeptical of this study because of the very small sample size. 11 people to begin with is small but to only gather data from 7 makes me wonder if there could be confounding or if the results are truly representative of other patients with HIV that only uses the CCR5 receptor. I figured that since it is a phase II clinical trial that they would need more participants to ensure that safety and accuracy of the drug. This study also raises more questions like what are the long term effects. Is this gene therapy something that will have to be done for the rest of a person's life? Is it a permanent solution? Does it halt the progression of disease and cause a patient to go into remission (low viral load, high CD4 count) or does it just stop the disease and the patient can still spread the disease on. Are there any side effects seen so far with gene therapy? These are some of the questions that popped in my head as I was reading this article. Is it possible to use gene therapy to have host cells recognize HIV's enzymes that way it can further prevent infection?

    Hopefully in the future other gene therapies will be developed to help other patients who have different strains of HIV. Especially in countries where they have an exceptionally high burden of HIV patients. This could be incredibly beneficial. I am also wondering if it is successful could this be the new standard of treatment.

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  70. Unfortunately my computer has been dysfunctional this week and I thought I had posted a response earlier.
    While gene therapy seems promising, only 3 out of 11 participants had long lasting effect due to the treatment. The experiment needs to be done on a more larger scale, generating more valuable data that could be more generalize-able to the population of HIV/AIDS patients.
    Also worthy of note was the participant who only experienced the effects of gene therapy for a limited amount of time. More research needs to be done to see how long the effects of gene therapy would last in a patient, whether they would require frequent doses, etc. Researchers should conduct more follow up sessions on the participants who had experienced favorable effects of the gene therapy to see if any changes occur in their status.

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  71. I believe that gene therapy is an option for people with HIV/AIDS. We have the ability to make major changes in a person’s DNA, and it’s highly possible that we can find a way to treat people with HIV with gene therapy. We are still in the early stages, so a lot more research needs to be completed. We must discover if there are any side effects or other problems that can occur from this therapy. We need a large sample size of individuals to test before we’re comfortable with our findings as well. The fact that there is evidence of making this a possibility is a huge step in treating patients with HIV/AIDS.

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