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

Understanding the molecular basis and pathogenesis of hereditary optic neuropathies: towards improved diagnosis and management - The Lancet Neurology

Frontiers In vivo Analysis of CRISPR/Cas9 Induced Atlastin Pathological Mutations in Drosophila

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

Oxidative stress monitoring in iPSC-derived motor neurons using genetically encoded biosensors of H2O2

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

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

Effective restoration of dystrophin expression in iPSC Mdx-derived muscle progenitor cells using the CRISPR/Cas9 system and homology-directed repair technology. - Abstract - Europe PMC

Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities, Journal of Nanobiotechnology

IJMS, Free Full-Text

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

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

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