RAN translation of C9orf72-related dipeptide repeat proteins in zebrafish recapitulates hallmarks of amyotrophic lateral sclerosis and identifies hypothermia as a therapeutic strategy
David Burrows, Alexander McGown, Olfat Abduljabbar, Lydia M. Castelli, Pamela J. Shaw, Guillaume M. Hautbergue, Tennore Ramesh
This study creates a zebrafish model of C9orf72-ALS/FTD using RAN translation and shows hypothermia reduces DPR pathology and motor deficits.
Annals of Neurology · Aug 31, 2024Peer-reviewedMolecular Biology