Can Gene Therapy Cure HIV?

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    Mar 06, 2014 7:06 PM GMT
    Promising...

    http://www.technologyreview.com/news/525286/can-gene-therapy-cure-hiv/

    Engineering a patient’s own immune cells to resist HIV could eliminate the need for lifelong antiretroviral therapies.

    The immune cells of HIV patients can be genetically engineered to resist infection, say researchers. In a small study in humans, scientists report that by creating a beneficial mutation in T cells, they may be able to nearly cure patients of HIV.

    In a study published in the New England Journal of Medicine on Wednesday, researchers report that they can use genome editing to re-create the rare mutations responsible for protecting about 1 percent of the population from the virus in infected patients. They report that some of the patients receiving the genome-modifying treatment showed decreased viral loads during a temporary halt of their antiretroviral drugs. In one patient, the virus could no longer be detected in his blood.

    Zinc-finger nucleases are one of a few genome-editing tools that researchers use to create specific changes to the genomes of living organisms and cells (see “Genome Surgery”). Scientists have previously used genome-editing techniques to modify DNA in human cells and nonhuman animals, including monkeys (see “Monkeys Modified with Genome Editing”). Now, the NEJM study suggests the method can also be safely used in humans.

    From each participating patient, the team harvested immune cells from the blood of the patients. They then used a zinc finger nuclease to “break” copies of the CCR5 gene that encodes for proteins on the surface of immune cells that are a critical entry point of HIV. The cells were then infused back into each patient’s bloodstream. The modification process isn’t perfect, so only some of the cells end up carrying the modification. “About 25 percent of the cells have at least one of the CCR5 genes interrupted,” says Edward Lanphier, CEO of Sangamo Biosciences, the Richmond, California, biotech company that manufactures zinc finger nucleases.

    Because the cells are a patient’s own, there is no risk of tissue rejection. The modified T cells are more resistant to infection by HIV, say the researchers.
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    Mar 06, 2014 9:11 PM GMT
    Gene manipulation will fix many, many, many things.
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    Mar 06, 2014 9:17 PM GMT
    beautiful!
  • Beeftastic

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    Mar 06, 2014 11:01 PM GMT
    This type of solution would likely need to be custom made for every patient, the cost would be prohibitive. It will be a long time before practical solutions could hit the marketplace, and they would still be hugely expensive.
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    Mar 07, 2014 2:22 PM GMT
    Beeftastic saidThis type of solution would likely need to be custom made for every patient, the cost would be prohibitive. It will be a long time before practical solutions could hit the marketplace, and they would still be hugely expensive.


    The tools for customized medicine are plummeting in price as technology improves. The other factor though is that you don't waste money on treatments that don't work - because of the increased sophistication of tests that help show what will work.

    There is a lot of speculation that over time this will actually be cheaper than conventional when you factor all the costs in (though these papers/articles are on other conditions):
    http://www.ucsf.edu/news/2014/02/111941/personalized-medicine-cost-effective-way-tailor-drug-therapy-after-stents (after stents)
    http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3041194/ (diabetes)