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

摘要

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 () or the RNA () repeats with therapeutic potential for several diseases. 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.) or the RNA (Batra et?al.) repeats with therapeutic potential for several diseases.