HIV Virus Used to Cure 2 Genetic Diseases. NAPSR Investigates.

WASHINGTON - Aug. 8, 2013 - PRLog -- Six children from all over the world, after three years of treatment, are well and show significant benefits. The announcement was made in two studies published today in Science by researchers at the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan, led by Luigi Naldini, demonstrating that gene therapy vectors derived from the HIV virus works against two severe genetic diseases, metachromatic leukodystrophy and Wiskott-Aldrich syndrome.

At the origin of both diseases is a genetic defect that results in the deficiency of a protein essential for the organism in the early years of life. In the case of metachromatic leukodystrophy, which currently lacks any effective treatment, it is the nervous system to be affected: babies with this disease are apparently healthy at birth, but at some point they begin to gradually lose the cognitive and motor skills they have acquired, with no possibility of arresting the neurodegenerative process. Children with Wiskott-Aldrich syndrome, on the other hand, have a faulty immune system that makes them much more vulnerable than normal to the development of infections, autoimmune diseases and cancer, as well as having a defect in the platelets which causes frequent bleeding.

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