Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes. Jiang X, Schaeffer L, Patni D, Russo T, et al. Science 2026;391:eadv2600.

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Amedeo Smart

Independent Medical Education

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Jiang X, Schaeffer L, Patni D, Russo T, et al. Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes. Science 2026;391:eadv2600.
PMID: 41643021