Microsatellite expansion RNA visualization, elimination, and reversal of molecular pathology by RNA-targeting Cas9

ABSTRACT: In this Study, we used RNA-targeting Cas9 (RCas9) to reverse characteristic Myotonic Dystrophy (DM1) cellular phenotypes such as elimination of RNA foci, MBNL relocalization, and reversal of transcriptome-wide splicing.

ORGANISM(S): Homo sapiens

PROVIDER: GSE100943 | GEO | 2017/08/10

SECONDARY ACCESSION(S): PRJNA393468

REPOSITORIES: GEO