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

Correction of the DYSF Gene in Miyoshi Myopathy

Cold shock' increases the frequency of homology directed repair gene editing in induced pluripotent stem cells

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

Rapid degeneration of iPSC-derived motor neurons lacking Gdap1 engages a mitochondrial-sustained innate immune response

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

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

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

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

Loss of OPA1 disturbs cellular calcium homeostasis and sensitizes for excitotoxicity

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

Dead Cas(t) light on new life: CRISPRa-mediated reprogramming of somatic cells into neurons