Voretigene neparvovec-rzyl (VN) therapy partially restored electroretinogram (ERG) responses in some RPE65-related retinopathy patients, with sustained improvements over several years. The study ...
CHICAGO — Children with a rare, inherited retinal disorder who were treated with the first Food and Drug Administration-approved gene therapy had noticeable improvements in vision that may be related ...
A new study shows that total RPE65 protein levels in mice with autosomal dominant retinitis pigmentosa were doubled following subretinal delivery of adeno-associated virus (AAV)-RPE65 gene ...
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed ...
Researchers have discovered profound similarities and surprising differences between humans and insects in the production of the critical light-absorbing molecule of the retina, 11-cis-retinal, also ...
Researchers report that deleting one of the inhibitors of the RPE65 gene in a mouse model that carries a human disease mutation prevents degeneration of cone photoreceptors that are used for daytime ...
In findings that could lead to curing some forms of congenital blindness through gene therapy, researchers at UCLA have discovered that RPE65, a gene missing in infants born with the blinding disease ...
LONDON and NEW YORK, Sept. 10, 2019 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that clinical data updates ...
Voretigene neparvovec (Luxturna; Novartis Pharmaceuticals UK) is an adeno-associated virus vector-based gene therapy. It introduces a healthy copy of the defective RPE65 gene into the retinal cells of ...
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