CRISPR/Cas9 Correction of OPA1 c.1334G>A: p.R445H Restores Mitochondrial Homeostasis in Dominant Optic Atrophy Patient-Derived iPSCs

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Researchers demonstrated generation of patient-derived OPA1 c.1334G>A: p.R445H mutant iPSCs, followed by correction of OPA1 through CRISPR/Cas9 guided homology directed repair and evaluated the effect of OPA1 correction on mitochondrial homeostasis.
[Molecular Therapy-Nucleic Acids]
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