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

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.

journal_name

Mol Cell

journal_title

Molecular cell

authors

Rao AN,Cooper TA

doi

10.1016/j.molcel.2017.10.023

subject

Has Abstract

pub_date

2017-11-02 00:00:00

pages

473-475

issue

3

eissn

1097-2765

issn

1097-4164

pii

S1097-2765(17)30798-0

journal_volume

68

pub_type

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