Fighting genetic disease with help of HIV virus

October 23, 2016

Boy treated with Gene Therapy for MLD

gene therapy trial in Italy led by Alessandra Biffi, MD, who now directs the Gene Therapy Program at Dana-Farber/Boston Children’s, has had promising results treating children with a devastating inherited neurological disorder called metachromatic leukodystrophy, CBS Sunday Morning reports.

The disease strikes seemingly healthy children who then begin to lose most of their capabilities and eventually succumb to the illness.


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