Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot–Marie–Tooth type 2A

Mitofusin-2 (MFN2) is an outer mitochondrial membrane protein essential for mitochondrial networking in most cells. Autosomal dominant mutations in the MFN2 gene cause Charcot–Marie–Tooth type 2A disease (CMT2A), a severe and disabling sensory-motor neuropathy that impacts the entire nervous system....

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Detalles Bibliográficos
Autores principales: Rizzo, Federica, Bono, Silvia, Ruepp, Marc David, Salani, Sabrina, Ottoboni, Linda, Abati, Elena, Melzi, Valentina, Cordiglieri, Chiara, Pagliarani, Serena, De Gioia, Roberta, Anastasia, Alessia, Taiana, Michela, Garbellini, Manuela, Lodato, Simona, Kunderfranco, Paolo, Cazzato, Daniele, Cartelli, Daniele, Lonati, Caterina, Bresolin, Nereo, Comi, Giacomo, Nizzardo, Monica, Corti, Stefania
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10676309/
https://www.ncbi.nlm.nih.gov/pubmed/38007410
http://dx.doi.org/10.1007/s00018-023-05018-w

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