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Gene Therapy for Leber Congenital Amaurosis | Hauswirth, William; Pang, Jijing; Boye, Sanford | The first AAV-mediated RPE gene therapy to restore visual function was obtained in a canine model of Leber Congenital Amaurosis (LCA), a severe form of autosomal recessive, childhood-onset retinal dystrophy 1;2. Humans with LCA are born with significant vision deficits and usually lose all vision wi... | 20070212_nanos_novelnotreatmentsympos_07.pdf |