• <em>RPGRIP1</em>-related retinal disease presenting as isolated cone dysfunction
• A subpopulation of activated retinal macrophages selectively migrated to regions of cone photoreceptor stress, but had limited effect on cone death in a mouse model for type 2 Leber congenital amaurosis
• AAV Serotypes and Their Suitability for Retinal Gene Therapy
• AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype
• AAV9 targets cone photoreceptors in the nonhuman primate retina
• Cellular and Molecular Mechanisms of Pathogenesis Underlying Inherited Retinal Dystrophies
• Challenges and Opportunities in the Genetic Analysis of Inherited Retinal Dystrophies in Africa, a Literature Review
• Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence
• Choroidal structure investigated by choroidal vascularity index in patients with inherited retinal diseases
• Clinical and Therapeutic Evaluation of the Ten Most Prevalent <em>CRB1</em> Mutations
• Comprehensive Genotyping and Phenotyping Analysis of GUCY2D-Associated Rod- and Cone-Dominated Dystrophies
• Cone Health and Retinoids
• Development of a novel prediction model based on protein structure for identifying RPE65-associated inherited retinal disease (IRDs) of missense variants
• Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in <em>CEP2</em>90-LCA: Replication in two eyes
• Evaluation of Ocular Gene Therapy in an Italian Patient Affected by Congenital Leber Amaurosis Type 2 Treated in Both Eyes
• Gene Therapy for Inherited RETINAL AND OPTIC NERVE Disorders: Current Knowledge
• Gene therapy for RPE65-related retinal disease
• Generation of two induced pluripotent stem cell lines (LVPEIi004-A and LVPEIi005-A) from probands with Leber Congenital Amaurosis 2 (LCA2) and harboring mutations in RPE65
• Genetic and Clinical Profile of Retinopathies Due to Disease-Causing Variants in Leber Congenital Amaurosis (LCA)-Associated Genes in a Large German Cohort
• Inherited retinal diseases in Germany-Challenges in health care supply structure and diagnostics
• Investor Outlook: Focus on Upcoming LCA2 Gene Therapy Phase III Results
• Leber Congenital Amaurosis / Early-Onset Severe Retinal Dystrophy Overview
• Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice
• PAM-flexible Engineered FnCas9 variants for robust and ultra-precise genome editing and diagnostics
• Psychometric Validation of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis
• RPE65 gene therapy slows cone loss in Rpe65-deficient dogs
• RPE65 is present in human green/red cones and promotes photopigment regeneration in an in vitro cone cell model
• SubILM Injection of AAV for Gene Delivery to the Retina
• The first gene therapy for <em>RPE65</em> biallelic dystrophy with voretigene neparvovec-rzyl in Brazil
• The rapidly evolving state of gene therapy
• Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2
• Voretigene Neparvovec for the Treatment of RPE65-associated Retinal Dystrophy: Consensus and Recommendations from the Korea RPE65-IRD Consensus Paper Committee