Gene therapy for Leber congenital amaurosis.
Dejneka
Nadine S
NS
University of Pennsylvania, Scheie Eye Institute, Philadelphia, PA 19104-6069, USA. ndejneka@mail.med.upenn.edu
Surace
Enrico M
EM
Bennett
Jean
J
D001766:Blindness / Q000151:congenital / Q000628:therapy*; D002352:Carrier Proteins; D005136:Eye Proteins; D015316:Genetic Therapy; D006801:Humans; D009154:Mutation; D029242:Optic Atrophy, Hereditary, Leber / Q000628:therapy*; D011506:Proteins / Q000235:genetics; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
D002352:Carrier Proteins; D005136:Eye Proteins; D011506:Proteins; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase