4.7 Editorial Material

Drug Screen Tugs at Common Thread for Repeat Disorders

Journal

TRENDS IN PHARMACOLOGICAL SCIENCES
Volume 41, Issue 2, Pages 71-73

Publisher

CELL PRESS
DOI: 10.1016/j.tips.2019.12.004

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Repeat-associated non-ATG (RAN) translation is emerging as a driver of pathogenesis in microsatellite expansion disorders. Green and colleagues recently identified several candidate RAN translation inhibitors from a high-throughput small-molecule screen for fragile X tremor ataxia syndrome. Their study establishes a path forward for identifying inhibitors of RAN translation for multiple disorders.

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