HI Virus as Gene Therapy

On July 11, 2013, Italian scientists who were able to use the HI virus as gene therapy to “cure” two rare genetic diseases, metachromatic leukodystrophy and Wiskott-Aldrich syndrome, published their studies in the journal Science. The study has been followed for three years in six children, three with one disease and three with the other. Both of these diseases are caused by a genetic defect that leads to deficiency of a protein essential to young children. This protein deficiency is found in the nervous system for children with metachromatic leukodystrophy, and in the immune system for children with Wiskott-Aldrich syndrome. The scientists took stem cells from the bone marrow of each patient and treated these cells so that they would be able to restore the missing protein. They recognized that they needed a viral vector, an agent that could deliver genetic material into cells, that could carry the corrected cells with the proteins. The viral vector from HIV was tested and proven to work, because after the altered stem cells carried by HIV viral vectors were reinjected into these patients’ bodies, the children have shown significant improvements.

            This is a fairly recent study that is receiving a lot of public recognition and will most likely continue to receive recognition for its use of an HIV viral vector. Even though this does not deal with addressing the needs of HIV patients, this is a phenomenon that may continue to be used in finding cures for other genetic diseases. Though these were rare genetic diseases, now that a solution has been found for both, if other children are to have either diseases in the future there is treatment for them. The fascinating thing about this is that not just one genetic disease was involved in this study, but two. And after the treatment, the children carrying either genetic disease were found to be healthier and showed less symptoms of the diseases. The scientists have sufficient evidence proving that using a HIV viral load has been effective in carrying corrected cells. When individuals think of HIV, it is a disease that many are trying to find the cure for and ideally would like to eliminate completely. However, by learning more about the virus and recognizing parts of the virus that could actually help with other research appears to be an efficient use of money and resources.

            A part that this research and study touches upon is the importance of stem cells and stem cell research. Another HIV story that was covered in 2013 talked about how two cases of HIV in patients were cured in two patients. After undergoing stem cell transplants, there was no trace of the HIV virus in their bodies. Possibly, through the research conducted by the Italian scientists in finding a cure for two genetic diseases, researchers trying to find a cure for HIV could use this research for their own research aiming to find a cure for HIV. Ideally, with more research done on HIV with a focus on stem cell research, scientists will eventually find a cure for HIV.

-Sierra Jeong

Sources: 

Image 1 (of HI virus particle): http://www.nature.com/news/gene-therapy-can-protect-against-hiv-1.9516

Image 2 (Stem Cell Research being conducted for HIV) : http://www.bloomberg.com/news/2013-07-03/stem-cell-transplants-clear-hiv-in-two-patients-in-study.html

News Article: http://health.usnews.com/health-news/news/articles/2013/07/11/scientists-use-hiv-to-cure-2-rare-genetic-diseases