Amelioration of Leigh syndrome induced by mouse blastocyst complementation with a mutant human mitochondrial ATP synthase 6

Abstract Background Mutation of the m. 8993 T > G ATP6 subunit of ATP synthase causes a maternally inherited Leigh Syndrome (LS), a rapidly fatal encephalomyelopathy in childhood called and related Neuropathy, Ataxia and Retinitis Pigmentosa (NARP) in adults. There is no cure, and relevant animal...

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Bibliographic Details
Main Authors: Huijun Yuan, Keith A. Webster, Muhammad Tariq Bhatti, William W. Hauswirth, Alfred S. Lewin, John Guy
Format: Article
Language:English
Published: Wiley 2022-09-01
Series:Clinical and Translational Discovery
Subjects:
Online Access:https://doi.org/10.1002/ctd2.114