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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...

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Detalles Bibliográficos
Autores principales: Corrà, Samantha, Cerutti, Raffaele, Balmaceda, Valeria, Viscomi, Carlo, Zeviani, Massimo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
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