Gene Therapy In HIV Infection

The various attempts to move closer to a cure for HIV have led scientists and researchers toward inventive new ways to approach the problem. The use of zinc finger nucleases (ZFNs) is one of those things. ZFNs disrupt the CCR5 receptor on the surface of CD4 cells. This receptor is what HIV uses to infect its host. Through the use of ZFNs, the current study attempts to answer the following hypothesis: could genetically altering a patient's CD4 cells using ZFNs result in making a patient's cells resistant to HIV? This is a breakthrough in HIV research and if successful, puts scientists one step closer to finding a functional cure for HIV.

There are actually people who are resistant to being infected with HIV, albeit a small percentage. It is a genetic mutation, called delta32, in the CCR5 receptor. A previous study discovered this mutation, and a CCR5 blocking drug called Maraviroc went into production. The current research goes even further than this: blocking HIV from entering CD4 cells entirely. An HIV-infected patient stricken with leukemia was given a stem cell transplant using a donor who had been born with delta32 mutation. The subsequent results were a huge discovery, to say the least.

The transplant, which is used in leukemia patients to create fresh white blood cells, had an additional benefit: it wiped out the HIV infection in the patient as well. Over 4 years later, the patient was still free of the disease, suggesting that he is cured completely. This is a remarkable discovery; one that should be talked about with great fanfare. However, it flew completely under the radar. While this isn't an indication that a cure has been found, it's good progress. Gene therapy in HIV infection still needs more testing done, as well as answering a few lingering questions.

There are several questions yet to be answered that the research study attempts to answer. How can this therapy help people who are already on an antiretroviral medication program? Can gene therapy benefit untreated HIV? Will the modified cells reduce the HIV viral load to a level low enough to allow a person on antiretroviral medication to stop taking it? How much will this procedure cost? All of these questions and more are still open, and more testing needs to be done. Before the word "cure" can be talked about, gene therapy research must continue and become more in-depth. The goal is HIV eradication, and gene therapy just puts the world one step closer to making that a reality.

The reference scientific workshop on HIV Persistence, HIV Reservoirs & Eradication Strategies: http://www.informedhorizons.com/persistence2011/

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HIV Cure: Gene Therapy Results Still Preliminary

HIV experts are optimistic about a recent study in which researchers were able to reconfigure blood cells, making them resistant to HIV. This trial was done on six HIV infected patients successfully treated with antiretroviral therapy (ART). But experts are not sure yet if this will actually be a cure to the deadly disease, though they all agree it is a step in the right direction. This study was done after a man in Berlin received a stem cell transplant and four years later is cured from HIV.

The HIV gene therapy results were announced in March 2011 during a medical conference in Boston, Massachusetts. This is a positive sign that there might be a cure found in the near future and will generate excitement in researchers to keep looking. This new approach will not completely eliminate HIV from the body, but it will make the virus controllable, most likely without the need of medication. It is called a functional cure.

An autologous cell transplant was performed in the study, where a human gene was permanently deleted and the altered cells were infused back into the patient. Previous gene therapies were to muffle the activity of a gene or add a new gene, both approaches where unsuccessful. Specifically, six men with HIV had some blood filtered and some T-Cells removed from that blood. A gene-snipping compound called 'zinc finger nuclease' was added to the T-cells. The modified cells were then mixed with growth factors before infusing them back into the patient's blood stream. Half of the men received about five billion modified cells and the other half received about 205 billion modified cells. After a year, these modified cells are still thriving and multiplying.

HIV attacks a person's T-cells, which are special immune system cells, through a protein receptor called CD4 and a coreceptor called CCR5. There are some people that lack the CCR5 gene and are naturally immune to HIV. When a person lacking the CCR5 gene donated bone marrow to a person with leukemia, who also happened to be HIV positive, the HIV infection was cured. This sparked the study. The patients had about two days worth of flu-like symptoms, which was the only reported side effect during the study. Not only is this a huge step in finding a cure for HIV and AIDS, but it is a huge revelation in the field of genetics. It is the first time in the history that doctors succeed to achieve a sabotage of the natural history of HIV infection.

The reference scientific workshop on HIV Persistence, HIV Reservoirs & Eradication Strategies: http://www.informedhorizons.com/persistence2011/

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