CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis | Science Advances
A New Gene Therapy For Early-onset RP
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS
RPE65 - Wikipedia
Gene Therapy & Clinical Trials to Treat Vision Loss | Fighting Blindness Canada
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis | Science Advances
RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy | Orphanet Journal of Rare Diseases | Full Text
Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population | Orphanet Journal of Rare Diseases | Full Text
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy: Molecular Therapy
Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3 - The Lancet
RPE65 Gene - GeneCards | RPE65 Protein | RPE65 Antibody
RPE65 gene therapy slows cone loss in Rpe65-deficient dogs | Gene Therapy
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement | European Journal of Human Genetics
IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials | British Journal of Ophthalmology
Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis: Molecular Therapy
A Gene Scan Study of RPE65 in Chinese Patients with Leber Congenital Amaurosis | Chinese Medical Journal
Retinal pigment epithelium 65 kDa protein (RPE65): An update - ScienceDirect
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS
A New Gene Therapy For Early-onset RP
Compound heterozygous RPE65 mutations associated with an early onset autosomal recessive retinitis pigmentosa - Owczarek‐Lipska - 2020 - The Journal of Gene Medicine - Wiley Online Library
RPE65 - an overview | ScienceDirect Topics
A Novel Mutation in the RPE65 Gene Causing Leber Congenital Amaurosis and Its Transcriptional Expression In Vitro | PLOS ONE
