Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

Since long before the first approval of gene therapy for retinal disease, ocular gene therapy
has captured the hopes of patients, clinicians, and scientists alike. Indeed, the retina …

Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital
deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic …

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) …

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

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

Cytosine base editors and adenine base editors (ABEs) can correct point mutations
predictably and independent of Cas9-induced double-stranded DNA breaks (which causes …

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 …