Drug Screen Tugs at Common Thread for Repeat Disorders

Kaalak Reddy; John Douglas Cleary; J Andrew Berglund

doi:10.1016/j.tips.2019.12.004

Drug Screen Tugs at Common Thread for Repeat Disorders

Trends Pharmacol Sci. 2020 Feb;41(2):71-73. doi: 10.1016/j.tips.2019.12.004. Epub 2020 Jan 8.

Authors

Kaalak Reddy¹, John Douglas Cleary¹, J Andrew Berglund²

Affiliations

¹ RNA Institute, State University of New York at Albany, Albany, NY, USA.
² RNA Institute, State University of New York at Albany, Albany, NY, USA; Department of Biological Sciences, State University of New York at Albany, Albany, NY, USA. Electronic address: aberglund@albany.edu.

PMID: 31926601
DOI: 10.1016/j.tips.2019.12.004

Abstract

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.

Keywords: RAN translation; RNA binding; multiple targets; therapeutic compound screen.

Publication types

Comment

MeSH terms

Ataxia / genetics
Fragile X Mental Retardation Protein* / genetics
Fragile X Mental Retardation Protein* / metabolism
Fragile X Syndrome* / drug therapy
Fragile X Syndrome* / genetics
Humans
Tremor / drug therapy
Tremor / genetics
Trinucleotide Repeat Expansion

Substances

Fragile X Mental Retardation Protein