RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
Alexandra Weiss, James W. Gilbert, Iris Valeria Rivera Flores, Jillian Belgrad, Chantal M. Ferguson, Elif O. Dogan, Nicholas Wightman, Kit Mocarski, Dimas Echeverria, Ashley L. Harkins, Ashley Summers, Brianna Bramato, Nicholas McHugh, Raymond C. Furgal, Nozomi Yamada, , Anastasia Khvorova
The paper shows that silencing SOD1 with optimized di-siRNA extends survival and prevents disease progression in ALS mice.
Molecular Therapy · May 9, 2025Peer-reviewedNeuroscience