Researchers at UCLA are working on ways to arm blood stem cells in the bone marrow against HIV.

The strategy is likely to be more effective than current anti-viral treatments and some day might have the potential to immunize people against the virus.

“If you can target the blood-forming stem cell, that cell gives rise to all blood cells,” said virologist Jerome Zack of UCLA. “So, therefore, if you could protect that cell, then every other cell derived from that would be protected.”

Zack presented his research Wednesday at a meeting of the Independent Citizens’ Oversight Committee, the 27-member group created by Proposition 71 to direct the state’s stem cell research program. The urgency to develop better treatments for HIV is particularly acute in San Francisco, where the disease has affected approximately 25 percent of the gay male population, said Robert Klein, chairman of the ICOC.

Zack said he hopes stem cell therapy could be a better strategy for fighting the virus. Zack is devising a way to insert a gene into bone marrow stem cells that can either prevent HIV from infecting the cells or deactivate any virus already in the cells.

The idea is to replace the gene that is vulnerable to attack by HIV with a synthetically engineered piece of DNA designed to seek out and destroy the virus. The DNA fragment, known as a ribozyme, is tailored specifically to bind to HIV and cut it in half, rendering it harmless.

Mitsuyasu recently finished an initial clinical trial to test the safety of the treatment. The 10 patients in the trial did not have any problems, and after three years, the HIV-resistant blood cells could still be detected.

“If this works, even though it may not cure the disease, it certainly would allow patients to go for periods of time without therapy,” Mitsuyasu said. “And that will make a big difference both in terms of the rate at which resistance develops to these medicines as well as all the side effects associated with having to take the medicine for the rest of their lives.”

Mitsuyasu is currently enrolling people in a trial to test the stem cell therapy.
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