HIV Helps Cure Rare Genetic Disorders

Italian scientists now report in the journal Science that they have used a piece of HIV to cure two rare genetic diseases that affect children.

Gene therapy using HIV to cure metachromic leukodystrophy and Wiskott Aldrich has proved effective in six children who endured three years of treatment. Metachromatic leukodystrophy, which affects the nervous system and causes a loss of mental and physical abilities, and Wiskott-Aldrich syndrome, which causes the immune system to malfunction, were both previously considered incurable.

In a press release from the San Raffaele-Telethon Institute for Gene Therapy, a joint project between two institutes in Milan, director Luigi Naldini said, “The results obtained from the first six patients are very encouraging: the therapy is not only safe, but also effective and able to change the clinical history of these severe diseases.”

In the new therapy, HIV serves as a viral vector to deliver a corrected copy of the defective gene into stem cells that have been taken from the patient’s bone marrow. Once reinjected into the body, the treated stem cells were able to restore the missing protein to key organs.

“The results described in these two studies make me very proud, both as a scientific director and as a pediatrician who has dedicated her entire professional life to children affected by genetic diseases,” said Maria Grazia Roncarolo of the San Raffaele Institute. “[They] give us great hope for the future of these children and for the possible cure of other genetic diseases.”