WP9 is dedicated to cardiovascular diseases, in this case in the context of skeletal muscle disease. Specific guide RNAs for CRISPR/Cas-based gene therapy of DMD will be developed and formulated into nanoparticles. Initially, the therapy will be studied experimentally in organoid models before final validation in large animal models. Since the dystrophin gene responsible for DMD is located on the X chromosome, WP9 also aims to activate the intact dystrophin allele on the second (inactive) X chromosome by manipulating epigenetic factors.