O.8DUX4 mRNA silencing with CRISPR-Cas13 gene therapy as a prospective treatment for Facioscapulohumeral muscular dystrophy

DUX4 de-repression causes Facioscapulohumeral muscular dystrophy (FSHD), which is associated with progressive muscle wasting and weakness that may lead to wheelchair dependence. There are currently no treatments that alter the course of FSHD and therapy development is an unmet need in the field. DUX4 genome editing using the CRISPR-Cas9 approach is promising but also has challenges, partly because the DUX4 gene is present in potentially hundreds of copies, embedded within identical or nearly identical D4Z4 repeats.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research