Article
Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A.
Cellular and molecular life sciences : CMLS - 25 Nov 2023
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
Abstract excerpt
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. Here, we propose a novel therapeutic strategy tailored to correcting the root genetic defect of CMT2A. Though...
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