Researchers from Stanford University have developed a method that allows them to copy and paste genes that would make immune cells resistant to HIV. The experimental treatment would prevent cells from being infected with HIV, and would allow already-infected HIV patients to resist the spiral into AIDS. The treatment could serve as welcome relief for the 34 million people worldwide infected with HIV, according to the World Health Organization, and for anyone who might consider themselves at risk for the disease.
The treatment is an escalation of the procedure that worked so well on the Berlin Patient. Named Timothy Hutter, the Berlin Patient had been diagnosed with AIDS and leukemia. Because some people are born with a mutation in the CCR5 receptor, one of the two proteins that HIV uses as an entryway into a T-cell, they are immune to HIV infection. Hutter received a bone marrow patient from a person with this mutation, and is now considered cured of HIV.
The treatment builds on that by copying and pasting HIV-resistant genes into a cell. There are two different methods currently being employed: scientists at Sangamo BioSciences in Richmond, California modified the CCR5 receptor so that it resembles the naturally resistant one. The company is currently conducting a trial on their method with AIDS patients.
The Stanford researchers found a perhaps even stronger method. They honed in on an undamaged section of the CCR5 receptor and inserted three genes that boost a person's immune resistance against HIV, preventing HIV's entryway via the CCR5 and CXCR4 receptors.
"Providing an infected person with resistant T cells would not cure their viral infection," said assistant professor at the University of Texas-Austin and co-author of the study Dr. Sara Sawyer in a statement. "However, it would provide them with a protected set of T cells that would ward off the immune collapse that typically gives rise to AIDS."
The treatment may spell the end of the antiretroviral cocktail of drugs that many HIV patients use in order to prevent the development of full-blown AIDS. The cocktail is necessary because HIV is famous for mutating, and the drugs attack HIV at various stages during the replication process.
Researchers are concerned though that the insertion of the new genes may provide a break elsewhere in the genome, causing cancer or other health concerns. They also worry that cells in some people will not respond to the addition and will not express the new proteins.
The research is just part of a latest trend of gene-tailored therapy. Researchers hope that they can tailor the same method to target other diseases, like sickle-cell anemia. They believe that they will go to trial with their technique in three to five years.
Published by Medicaldaily.com