Leber congenital amaurosis (LCA) is a rare hereditary retinal degeneration caused by
mutations in more than a dozen genes. RPE65, one of these mutated genes, is highly …

Leber congenital amaurosis (LCA) associated with retinal pigment epithelium-specific
protein 65 kDa (RPE65) mutations is a severe hereditary blindness resulting from both …

Purpose To provide an initial assessment of the safety of a recombinant adeno-associated
virus vector expressing RPE65 (rAAV2-CB-hRPE65) in adults and children with retinal …

Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal
diseases that are incurable. One molecular form is caused by mutations in the RPE65 …

purpose. To characterize the spectrum of RPE65 mutations present in 453 patients with
retinal dystrophy with an interest in understanding the range of functional deficits attributable …

Leber congenital amaurosis (LCA) is a group of monogenic inherited retinal degenerations
that typically show early onset and severe visual dysfunction. In addition, there is a natural …

Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal
pigment epithelium–specific 65-kD protein (RPE65) is associated with poor vision at birth …

purpose. To quantify the residual vision in Leber congenital amaurosis (LCA) caused by
RPE65 mutations. methods. Patients with RPE65-LCA (n= 30; ages, 4–55) were studied …

Objective To determine the safety and efficacy of subretinal gene therapy in the RPE65 form
of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) …

The form of hereditary childhood blindness Leber congenital amaurosis (LCA) caused by
biallelic RPE65 mutations is considered treatable with a gene therapy product approved in …