CDK12 inhibition reduces abnormalities in cells from patients with myotonic dystrophy and in a mouse model

Authors: Ami Ketley, Marzena Wojciechowska, Sonja Ghidelli-Disse, Paul Bamborough, Tushar K. Ghosh, Marta Lopez Morato, Saam Sedehizadeh, Naveed Altaf Malik, Zhenzhi Tang, Paulina Powalowska, Matthew Tanner, Rudolf Billeter-Clark, Rebecca C. Trueman, Philippine C. Geiszler, Alessandra Agostini, Othman Othman, Markus Bösche, Marcus Bantscheff, Martin Rüdiger, Danuta E. Mossakowska, David H. Drewry, William J. Zuercher, Charles A. Thornton, Gerard Drewes, Iain Uings, Christopher J. Hayes, J. David Brook

Published: 2020-04-29

DOI: 10.1126/scitranslmed.aaz2415

Source: Full article


Abstract

CDK12 inhibition reduces mutant transcripts and nuclear foci in DM1 cells and produces splicing correction and phenotypic benefit in a mouse model.