RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
RNAi介导的SOD1基因沉默显著延长了ALS小鼠的生存期并改善了其功能预后。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.05.010
Weiss, Alexandra; Gilbert, James W; Rivera Flores, Iris Valeria; Belgrad, Jillian; Ferguson, Chantal; Dogan, Elif O; Wightman, Nicholas; Mocarski, Kit; Echeverria, Dimas; Harkins, Ashley L; Summers, Ashley; Bramato, Brianna; McHugh, Nicholas; Furgal, Raymond; Yamada, Nozomi; Cooper, David; Monopoli, Kathryn; Godinho, Bruno M D C; Hassler, Matthew R; Yamada, Ken; Greer, Paul; Henninger, Nils; Brown, Robert H Jr; Khvorova, Anastasia