Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A
以 MFN2 为靶点的 RNA 干扰和基因替代疗法联合治疗作为治疗 2A 型 Charcot-Marie-Tooth 病的原理证明
期刊:Cellular and Molecular Life Sciences
影响因子:6.2
doi:10.1007/s00018-023-05018-w
Federica Rizzo, Silvia Bono, Marc David Ruepp, Sabrina Salani, Linda Ottoboni, Elena Abati, Valentina Melzi, Chiara Cordiglieri, Serena Pagliarani, Roberta De Gioia, Alessia Anastasia, Michela Taiana, Manuela Garbellini, Simona Lodato, Paolo Kunderfranco, Daniele Cazzato, Daniele Cartelli, Caterina