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1 Gene Therapy for Leber Congenital AmaurosisHauswirth, William; Pang, Jijing; Boye, SanfordThe 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
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