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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Last updated 21 abril 2025


CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease

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Melatonin Repairs Osteoporotic Bone Defects in Iron-Overloaded

PDF) Modelling autosomal dominant optic atrophy associated with

CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores

PDF) Modelling autosomal dominant optic atrophy associated with

CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores

Functional genomics and the future of iPSCs in disease modeling

Biomedicines, Free Full-Text

CRISPR/Cas9-mediated A4GALT suppression rescues Fabry disease

CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores

Characterisation of a novel OPA1 splice variant resulting in
Gene regulation analysis of patient-derived iPSCs and its CRISPR
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