The consortium project to treat RPE65 deficiency in humans.
Hauswirth
William W
WW
Ophthalmology and Molecular Genetics at the University of Florida School of Medicine, Gainesville, FL, USA.
D000818:Animals; D001766:Blindness / Q000235:genetics / Q000628:therapy*; D002352:Carrier Proteins; D017321:Clinical Trials, Phase I as Topic; D000229:Dependovirus / Q000235:genetics*; D015785:Eye Diseases, Hereditary / Q000235:genetics / Q000628:therapy*; D005136:Eye Proteins / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D035583:Rare Diseases / Q000235:genetics / Q000628:therapy; D012164:Retinal Diseases / Q000235:genetics / Q000628:therapy*; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
D002352:Carrier Proteins; D005136:Eye Proteins; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase