Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy: Molecular Therapy
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
Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis: Molecular Therapy
Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials | British Journal of Ophthalmology
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS
RPE65 gene therapy slows cone loss in Rpe65-deficient dogs | Gene Therapy
RPE65-related retinal dystrophy: mutational and phenotypic spectrum in 45 affected patients | medRxiv
RPE65 - Wikipedia
Luxturna: Gene Therapy for Biallelic RPE65 Mutation-Associated Retinal Dystrophy - YouTube
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis | Science Advances
IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
Sequence analysis of the RPE65 gene missense mutation Leu67Arg... | Download Scientific Diagram
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement | European Journal of Human Genetics
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy: Molecular Therapy
Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population | Orphanet Journal of Rare Diseases | Full Text
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
RPE65 mutation frequency and phenotypic variation according to exome sequencing in a tertiary centre for genetic eye diseases in China - Li - 2020 - Acta Ophthalmologica - Wiley Online Library
RPE65 - Wikipedia
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis | Science Advances
A Novel Mutation in the RPE65 Gene Causing Leber Congenital Amaurosis and Its Transcriptional Expression In Vitro | PLOS ONE
A Novel Mutation in the RPE65 Gene Causing Leber Congenital Amaurosis and Its Transcriptional Expression In Vitro | PLOS ONE
A New Gene Therapy For Early-onset RP
IJMS | Free Full-Text | Inherited Retinal Diseases Due to RPE65 Variants: From Genetic Diagnostic Management to Therapy
Cells | Free Full-Text | The Predicted Splicing Variant c.11+5G>A in RPE65 Leads to a Reduction in mRNA Expression in a Cell-Specific Manner
RPE65 - retinal degeneration: for patients - Gene Vision
RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy | Orphanet Journal of Rare Diseases | Full Text
A New Gene Therapy For Early-onset RP
Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark | European Journal of Human Genetics
