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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2021
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| Materias: | |
| 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 |