CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs
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
Abstract
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Topics & Concepts
CRISPRAtrophyBiologyHomeostasisMitochondrial diseaseInduced pluripotent stem cellNeuroscienceGeneticsMedicineMitochondrial DNACell biologyGeneEmbryonic stem cellMitochondrial Function and PathologyATP Synthase and ATPases ResearchCerebrovascular and genetic disorders