A Novel Mutation in the RPE65 Gene Causing Leber Congenital Amaurosis and Its Transcriptional Expression In Vitro | PLOS ONE
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS
Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3 - The Lancet
RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy | Orphanet Journal of Rare Diseases | Full Text
Get to know RPE65 - Luxturna HCP
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS
RPE65 - retinal degeneration: for patients - Gene Vision
![Figure 1, [Visual Cycle]. - CADTH Issues in Emerging Health Technologies - NCBI Bookshelf](https://www.ncbi.nlm.nih.gov/books/NBK538375/bin/eh0067_voretigene_neparvovec_emerging_healthf1.jpg "Figure 1, [Visual Cycle]. - CADTH Issues in Emerging Health Technologies - NCBI Bookshelf")
Figure 1, [Visual Cycle]. - CADTH Issues in Emerging Health Technologies - NCBI Bookshelf
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis | Science Advances
Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population | Orphanet Journal of Rare Diseases | Full Text
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement | European Journal of Human Genetics
Genes | Free Full-Text | Properties and Therapeutic Implications of an Enigmatic D477G RPE65 Variant Associated with Autosomal Dominant Retinitis Pigmentosa
IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial - The Lancet
Let's Talk About Gene Therapy for Inherited Retinal Diseases - Retina Today
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
Retinal pigment epithelium 65 kDa protein (RPE65): An update - ScienceDirect
What is LUXTURNA? - LUXTURNA® (voretigene neparvovec-rzyl)
Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark | European Journal of Human Genetics
RPE65-related retinal dystrophy: mutational and phenotypic spectrum in 45 affected patients | medRxiv
RPE65 gene therapy slows cone loss in Rpe65-deficient dogs | Gene Therapy
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy: Molecular Therapy
Frontiers | Gene Therapy to the Retina and the Cochlea
RPE65 - an overview | ScienceDirect Topics
