CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs: Molecular Therapy - Nucleic Acids
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CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease
Melatonin Repairs Osteoporotic Bone Defects in Iron-Overloaded
CRISPR/Cas9 therapeutics: progress and prospects
Enhanced genome editing in human iPSCs with CRISPR-CAS9 by co
CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease
PDF) Optimized OPA1 Isoforms 1 and 7 Provide Therapeutic Benefit
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Functional genomics and the future of iPSCs in disease modeling
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