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

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第一作者： Paul E,Sladen

第一单位： UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.

作者： Paul E,Sladen ^[1] ; Pedro R L,Perdigão ^[1] ; Grace,Salsbury ^[2] ; Tatiana,Novoselova ^[2] ; Jacqueline,van der Spuy ^[1] ; J Paul,Chapple ^[2] ; Patrick,Yu-Wai-Man ^[3] ; Michael E,Cheetham ^[1]

作者单位： UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK. ^[1] Centre for Endocrinology, William Harvey Research Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, Charterhouse Square, London EC1M 6BQ, UK. ^[2] UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.;Moorfields Eye Hospital NHS Foundation Trust, London EC1V 2PD, UK.;Cambridge Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, ED Adrian Building, Robinson Way, Cambridge CB2 0PY, UK.;John van Geest Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK. ^[3]

关键词 CRISPR OPA1 apoptosis bioenergetics gene correction gene editing iPSC mitochondria optic atrophy retinal ganglion cell

DOI 10.1016/j.omtn.2021.08.015

PMID 34589289

发布时间 2024-04-04

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