Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy

Authors: Angela Lek, Yuanfan Zhang, Keryn G. Woodman, Shushu Huang, Alec M. DeSimone, Justin Cohen, Vincent Ho, James Conner, Lillian Mead, Andrew Kodani, Anna Pakula, Neville Sanjana, Oliver D. King, Peter L. Jones, Kathryn R. Wagner, Monkol Lek, Louis M. Kunkel

Published: 2020-03-25

DOI: 10.1126/scitranslmed.aay0271

Source: Full article


Abstract

Genome-wide CRISPR-Cas9 screens identify druggable pathways associated with facioscapulohumeral muscular dystrophy.