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

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

SURF1 (surfeit locus protein 1)-related Leigh syndrome is an early-onset neurodegenerative disorder, characterized by reduction in complex IV activity, resulting in disrupted mitochondrial function. Currently, there are no treatment options available. To test our hypothesis that adeno-associated vir...

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Bibliographic Details
Main Authors: Qinglan Ling, Matthew Rioux, Yuhui Hu, MinJae Lee, Steven J. Gray
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
SURF1
Leigh syndrome
gene therapy
adeno-associated virus
AAV
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001364

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http://www.sciencedirect.com/science/article/pii/S2329050121001364

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