Gene Therapy Restores Sight

September 26, 2008

The individuals — their identities remain confidential — are participants in an early-stage clinical trial of gene therapy for Leber’s Congenital Amaurosis, a rare and untreatable form of congenital blindness.

Though the trial was designed to test the therapy’s safety rather than its efficacy, its benefits were so impressive that the researchers decided to publicize their results. (Wired)