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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 Ferguson,Elif O Dogan,Nicholas Wightman, Kit Mocarski,Dimas Echeverria, Ashley L Harkins,Ashley Summers,Brianna Bramato,Nicholas McHugh,Raymond Furgal, Nozomi Yamada,David Cooper,Kathryn Monopoli,Bruno M D C Godinho,Matthew R Hassler,Ken Yamada,Paul Greer,Nils Henninger,Robert H Brown,Anastasia Khvorova

Molecular therapy the journal of the American Society of Gene Therapy（2025）

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