A Therapeutic Double Whammy: Transcriptional or Post-transcriptional Suppression of Microsatellite Repeat Toxicity by Cas9
2017
Rao, Ashish N. | Cooper, Thomas A.
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.
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