A Therapeutic Double Whammy: Transcriptional or Post-transcriptional Suppression of Microsatellite Repeat Toxicity by Cas9

Ashish N Rao; Thomas A Cooper

doi:10.1016/j.molcel.2017.10.023

A Therapeutic Double Whammy: Transcriptional or Post-transcriptional Suppression of Microsatellite Repeat Toxicity by Cas9

Mol Cell. 2017 Nov 2;68(3):473-475. doi: 10.1016/j.molcel.2017.10.023.

Authors

Ashish N Rao¹, Thomas A Cooper²

Affiliations

¹ Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX 77030, USA; Department of Pathology and Immunology, Baylor College of Medicine, Houston, TX 77030, USA.
² Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX 77030, USA; Department of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX 77030, USA; Department of Pathology and Immunology, Baylor College of Medicine, Houston, TX 77030, USA. Electronic address: tcooper@bcm.edu.

PMID: 29100050
DOI: 10.1016/j.molcel.2017.10.023

Abstract

Microsatellite expansion diseases are caused by unstable tandem repeats of 3-10 nucleotides that become pathogenic beyond a threshold number of copies. Two groups present different approaches to reduce pathogenesis by targeting deactivated Cas9 to either the DNA (Pinto et al., 2017) or the RNA (Batra et al., 2017) repeats with therapeutic potential for several diseases.

Publication types

Comment

MeSH terms

Animals
CRISPR-Cas Systems*
Horses
Microsatellite Repeats
RNA*

Substances

RNA