Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy
Darina Šikrová, Vlad A. Cadar, Yavuz Ariyürek, Jeroen F. J. Laros, Judit Balog, Silvère M. van der Maarel
Abstract
polyadenylation signal as a therapeutic target and represent the first step toward clinical application of the CRISPR-Cas9 base editing platform for FSHD gene therapy.
Topics & Concepts
Facioscapulohumeral muscular dystrophyBiologySkeletal muscleChromatinPolyadenylationMuscular dystrophyGeneticsSomatic cellCell biologyGeneMessenger RNAAnatomyCRISPR and Genetic EngineeringRNA Research and SplicingMuscle Physiology and Disorders