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Harvard Stem Cell Institute (HSCI) researchers at Massachusetts General (MGH) and Boston Children’s hospitals (BCH) for the first time have used a relatively new gene-editing technique to create what could prove to be an effective technique for blocking HIV from invading and destroying patients’ immune systems.

The technology efficiently and precisely edits clinically relevant genes out of cells collected directly from people, in this case human blood forming stem cells and T-cells.

Though the researchers that believe this new approach to HIV therapy might be ready for human safety trials in less than five years, they themselves offered three strong points of caution:

The first and most obvious is that they could run into unexpected complications; the second is that the history of the HIV/AIDS epidemic is littered with “cures” that turned out not to be; and finally, even if this new approach works perfectly, it will require additional developments to be applicable in the areas of the world that have been the hardest hit by the epidemic.

The work, led by Chad Cowan, and Derrick Rossi, associate professors in Harvard’s Department of Stem Cell and Regenerative Biology, is featured on the cover of this month’s issue of the journal Cell Stem Cell.

David Scadden, a hematologist/oncologist who is both co-director of HSCI and director of the Center for Regenerative Medicine at MGH, called the new work “a tremendous first step in editing out what makes human cells vulnerable to HIV. It makes possible the idea that a person’s own immune cells can attack HIV without being susceptible to it. Since this was done in stem cells, the entire immune system may be durably brought to bear on the virus. That’s a powerful concept.

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