Italian researchers reported that they effectively eliminated the symptoms of children with two rare genetic diseases using gene therapy that involved a modified HIV-derived virus. The two clinical trials, published in the journal Science, focused on three children who were born with metachromatic leukodystrophy and three others with Wiskott-Aldrich syndrome. The virus, which was engineered so it could not cause AIDS, was attached with the normal version of the faulty gene found in each disease and delivered to bone marrow stem cells that were then transfused into the patients.
Bioengineered HIV virus treats genetic disease symptoms in studies
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