RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Weiss A, Gilbert JW, Rivera Flores IV, Belgrad J, Ferguson C, Dogan EO, Wightman N, Mocarski K, Echeverria D, Harkins AL, Summers A, Bramato B, McHugh N, Furgal R, Yamada N, Cooper D, Monopoli K, Godinho BMDC, Hassler MR, Yamada K, Greer P, Henninger N, Brown RH Jr, Khvorova A.
Weiss A, et al. Among authors: summers a.
Mol Ther. 2025 May 9:S1525-0016(25)00380-6. doi: 10.1016/j.ymthe.2025.05.010. Online ahead of print.
Mol Ther. 2025.
PMID: 40349108
Free article.