Due to improved phenoty** and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …

The recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65
mutation-associated inherited retinal dystrophy with viable retinal cells represents an …

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) …

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 …

The RPE65 gene encodes the isomerase of the retinoid cycle, the enzymatic pathway that
underlies mammalian vision. Mutations in RPE65 disrupt the retinoid cycle and cause a …

Widespread gene transfer to the retina is challenging as it requires vector systems to
overcome physical and biochemical barriers to enter and diffuse throughout retinal tissue …

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 …

Recombinant adeno-associated virus (rAAV) vectors possess a number of properties that
may make them suitable for clinical gene therapy, including being based upon a virus for …

Nonsyndromic recessive retinal dystrophies cause severe visual impairment due to the
death of photoreceptor and retinal pigment epithelium cells. These diseases until recently …

Severe inherited retinal diseases, such as retinitis pigmentosa and Leber congenital
amaurosis, are caused by mutations in genes preferentially expressed in photoreceptors …