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

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

Autosomal dominant optic atrophy (DOA) is the most common inherited optic neuropathy in the United Kingdom. DOA has an insidious onset in early childhood, typically presenting with bilateral, central visual loss caused by the preferential loss of retinal ganglion cells. 60%–70% of genetically confir...

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Bibliographic Details
Main Authors: Paul E. Sladen, Pedro R.L. Perdigão, Grace Salsbury, Tatiana Novoselova, Jacqueline van der Spuy, J. Paul Chapple, Patrick Yu-Wai-Man, Michael E. Cheetham
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
mitochondria
gene editing
CRISPR
gene correction
iPSC
optic atrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121002092

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http://www.sciencedirect.com/science/article/pii/S2162253121002092

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