Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome

Author:

Ling QinglanORCID,Rioux MatthewORCID,Hu Yuhui,Lee MinJaeORCID,Gray Steven J.ORCID

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference36 articles.

1. Leigh syndrome with COX deficiency and SURF1 gene mutations: MR imaging findings;Rossi;AJNR Am. J. Neuroradiol.,2003

2. Leigh syndrome: One disorder, more than 75 monogenic causes;Lake;Ann. Neurol.,2016

3. SURF1, encoding a factor involved in the biogenesis of cytochrome c oxidase, is mutated in Leigh syndrome;Zhu;Nat. Genet.,1998

4. Mutations of SURF-1 in Leigh disease associated with cytochrome c oxidase deficiency;Tiranti;Am. J. Hum. Genet.,1998

5. Surf1, associated with Leigh syndrome in humans, is a heme-binding protein in bacterial oxidase biogenesis;Bundschuh;J. Biol. Chem.,2009

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