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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Detalles Bibliográficos
Autores principales: Sladen, Paul E., Perdigão, Pedro R.L., Salsbury, Grace, Novoselova, Tatiana, van der Spuy, Jacqueline, Chapple, J. Paul, Yu-Wai-Man, Patrick, Cheetham, Michael E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8455316/
https://www.ncbi.nlm.nih.gov/pubmed/34589289
http://dx.doi.org/10.1016/j.omtn.2021.08.015

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8455316/
https://www.ncbi.nlm.nih.gov/pubmed/34589289
http://dx.doi.org/10.1016/j.omtn.2021.08.015

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