Sunday, October 23, 2016

Gene Therapy Using Vector Derived from HIV

According to CBS News, Italian researchers are using vectors derived from HIV in gene therapy for late infantile metachromatic leukodystrophy (MLD), which is a genetic disorder characterized by degeneration of white matter of the brain and central nervous system. People with this disease lack the enzyme arylsulfatase-A and cannot break down sulfatides, which then accumulate in the white matter and cause demyelination (myelin sheaths are necessary for normal neural transmission). Most children with the late infantile form die by age 5, and there is currently no FDA-approved cure.

However, a clinical trial in Milan, Italy is introducing the wild-type version of the gene into patients using vectors derived from HIV. The scientists chose HIV because of its efficiency at entering cells. The treatment is used on children who have not developed symptoms. So far, at least 70-80% of the patients have had positive results and are able to attend school. They return to Milan for testing and monitoring every 6 months. Only 24 patients in the world have received this therapy, and the trial is not accepting new participants.

-Sally Tran


CBS News. "Fighting Genetic Disorder with Help from HIV. " 2016 Oct 23.

Info about MLD:.

National Institute of Neurological Disorders and Stroke. “NINDS Metachromatic Leukodystrophy Information Page.” 2016 Feb 22.

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