OPA1 gene therapy prevents retinal ganglion cell loss in a Dominant Optic Atrophy mouse model

Dominant optic atrophy (DOA) is a rare progressive and irreversible blinding disease which is one of the most frequent forms of hereditary optic neuropathy. DOA is mainly caused by dominant mutation in the OPA1 gene encoding a large mitochondrial GTPase with crucial roles in membrane dynamics and ce...

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發表在:Sci Rep
Main Authors: Sarzi, Emmanuelle, Seveno, Marie, Piro-Mégy, Camille, Elzière, Lucie, Quilès, Mélanie, Péquignot, Marie, Müller, Agnès, Hamel, Christian P., Lenaers, Guy, Delettre, Cécile
格式: Artigo
語言:Inglês
出版: Nature Publishing Group UK 2018
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5802757/
https://ncbi.nlm.nih.gov/pubmed/29410463
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-018-20838-8
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