A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4 Mice

Title
A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4 Mice
Authors
Keywords
mitochondrial diseases, gene therapy, AAV, Ndufs4, complex I, OXPHOS, PHP.B, Leigh syndrome
Journal
Molecular Therapy-Methods & Clinical Development
Volume 17, Issue -, Pages 1071-1078
Publisher
Elsevier BV
Online
2020-05-05
DOI
10.1016/j.omtm.2020.04.026

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