CRISPR/dCas9-mediated tuning of DMPK transcription reveals a quantitative relationship between toxic repeat RNA expression and MBNL1 activity in myotonic dystrophy. (academic.oup.com)

Authors: Ripken L … Wansink DG Journal: Human Molecular Genetics Published: 2026-08-10 DOI: https://doi.org/10.1093/hmg/ddag079