Gene Therapy for HIV Delivers Hopeful Results – US News

Posted: March 7, 2014 at 1:41 am

After having the genetic material in their white blood cells modified, some HIV patients saw their virus levels drop.

Gene editing a process used in a recent study targeting HIV is precisely what it sounds like and may hold new hope for patients battling the virus that causes AIDS.

Researchers involved in the study removed white blood cells the soldiers of the bodys immune system from the blood of12 HIV patients, then cut into the genes in those cellsand sewed in new pieces of DNA. They thengrew the modified immune cells and reinserted them into the patients.

[READ:Genetic Mutations Could Shrink Risk of Type 2 Diabetes]

The modified cells blocked the virus from fulfilling its mission: hijacking the immune system and leaving patients susceptible to myriad diseases.

"This study shows that we can safely and effectively engineer an HIV patient's own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs," Dr. Carl June, a study author and a professor in immunotherapy at theUniversity of Pennsylvanias Perelman School of Medicine, said in a release.

In a sense, what the study tests is the ability to trick HIV. By modifying a gene called CCR5 that contains the code fora receptor that, when present, essentiallyopens the door of a cellto the virus, researchers aim to prevent the virus from spreading.

Researchers at Penn Medicine and Yeshiva University's Albert Einstein College of Medicine gave each of 12 patients a single infusion of roughly 10 billion modified cells between May 2009 and July 2012. Sangamo BioSciences, a California-based biopharmaceutical company, developed the technology for the therapy and donated the genetic material that was stitched into patients' genes.

After a month, half of the patients were taken off their traditional HIV treatments for 12 weeks, and four saw their virus levels drop. Onepatients viral load dropped below detection levels, and researchers discovered this patient already had a naturally occurring mutation similar to the therapy.

"Since half the subject's CCR5 genes were naturally disrupted, the gene editing approach was building on the head start provided by inheriting the mutation from one parent," saidstudy author Bruce Levine, aUniversity of Pennsylvaniaprofessor in cancer gene therapy.

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Gene Therapy for HIV Delivers Hopeful Results - US News

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