CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs - ScienceDirect

CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease phenotypes in a kidney organoid model - ScienceDirect

PDF) CRISPR/Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in Dominant Optic Atrophy patient-derived iPSCs

CRISPR editing demonstrates rs10490924 raised oxidative stress in iPSC- derived retinal cells from patients with ARMS2/HTRA1-related AMD

Function and Immunogenicity of Gene-corrected iPSC-derived Hepatocyte-Like Cells in Restoring Low Density Lipoprotein Uptake in Homozygous Familial Hypercholesterolemia

Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases: Molecular Therapy

High-throughput phenotypic screen for genetic modifiers in patient-derived OPA1 mutant fibroblasts identifies PGS1 as a functional suppressor of mitochondrial fragmentation

CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa: Molecular Therapy

CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease phenotypes in a kidney organoid model - ScienceDirect

CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease phenotypes in a kidney organoid model - ScienceDirect

CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease phenotypes in a kidney organoid model - ScienceDirect

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CRISPR/Cas9-Mediated Gene Correction in Osteopetrosis Patient-Derived iPSCs

A recurrent deletion mutation in OPA1 causes autosomal dominant optic atrophy in a Chinese family