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Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes.

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Bibliographic Details
Title: Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes.
Authors: Jiang, Xin1,2 (AUTHOR), Schaeffer, Laure3 (AUTHOR), Patni, Divya1 (AUTHOR), Russo, Tommaso1,4 (AUTHOR), Lee, Chao-Zong1,2 (AUTHOR), Aguilar, Corey1,2 (AUTHOR), Marques, Christine1 (AUTHOR), Jansen-West, Karen5 (AUTHOR), Hruska-Plochan, Marian6 (AUTHOR), Ray-Soni, Ananya1,2 (AUTHOR), Lim, Su Min1,2 (AUTHOR), Held, Aaron1 (AUTHOR), Yue, Mei5 (AUTHOR), Castellanos Otero, Paula5 (AUTHOR), Aryal, Sandeep1,2 (AUTHOR), Beaussant, Hortense D. A. M.1 (AUTHOR), Basu, Himanish7 (AUTHOR), Takakuwa, Hiro8 (AUTHOR), Daughrity, Lillian M.5 (AUTHOR), Ramesh, Nandini1,2 (AUTHOR)
Source: Science. 2/5/2026, Vol. 391 Issue 6785, p1-17. 17p.
Database: Academic Search Ultimate
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Description
ISSN:00368075
DOI:10.1126/science.adv2600