Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes.
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Jiang X, et al. Among authors: jain a.
Science. 2026 Feb 5;391(6785):eadv2600. doi: 10.1126/science.adv2600. Epub 2026 Feb 5.
Science. 2026.
PMID: 41643021
Free PMC article.