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Double administration of self-complementary AAV9(NDUFS4) prevents Leigh disease in Ndufs4(−/−) mice
Leigh disease, or subacute necrotizing encephalomyelopathy, a genetically heterogeneous condition consistently characterized by defective mitochondrial bioenergetics, is the most common oxidative-phosphorylation related disease in infancy. Both neurological signs and pathological lesions of Leigh di...
Autores principales: | Corrà, Samantha, Cerutti, Raffaele, Balmaceda, Valeria, Viscomi, Carlo, Zeviani, Massimo |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9586549/ https://www.ncbi.nlm.nih.gov/pubmed/36270002 http://dx.doi.org/10.1093/brain/awac182 |
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