'Defanged' HIV used to deliver gene therapy

Jul 15 2013

Researchers are having success using a "defanged" version of HIV to deliver gene therapy to children suffering from genetic diseases, a development that could improve other such care.

The Wall Street Journal: Defanged HIV Shows Promise In Gene Therapy 
Six children with rare genetic diseases were successfully treated using gene therapy that was delivered with a modified form of the AIDS virus, researchers said Thursday. HIV, which causes AIDS, is proving a boon to gene therapy because the ability to infect cells that makes the virus so dangerous has also rendered it an efficient agent for delivering replacement genes into a patient (Winslow, 7/11).

Los Angeles Times: Gene Therapy Using HIV Helps Children With Fatal Diseases, Study Says
Italian researchers have used a defanged version of HIV to replace faulty genes -- and eliminate devastating symptoms -- in children suffering two rare and fatal genetic diseases. Improved gene therapy techniques prevented the onset of metachromatic leukodystrophy in three young children and halted the progression of Wiskott-Aldrich syndrome in three others (Pandika, 7/11).