Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model

Autor: Bersani, Margherita, Rizzuti, Mafalda, Pagliari, Elisa, Garbellini, Manuela, Saccomanno, Domenica, Moulton, Hong M., Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania, Nizzardo, Monica
Zdroj: In Molecular Therapy 2 March 2022 30(3):1288-1299
Databáze: ScienceDirect