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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Detalles Bibliográficos
Autores principales: Ling, Qinglan, Rioux, Matthew, Hu, Yuhui, Lee, MinJae, Gray, Steven J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8517205/
https://www.ncbi.nlm.nih.gov/pubmed/34703839
http://dx.doi.org/10.1016/j.omtm.2021.09.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8517205/
https://www.ncbi.nlm.nih.gov/pubmed/34703839
http://dx.doi.org/10.1016/j.omtm.2021.09.001

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