CRISPR Therapeutics DM1 program

CRISPR Therapeutics' DM1 program is an investigational CRISPR/Cas9-based gene editing therapy being developed in collaboration with Vertex Pharmaceuticals for the treatment of Myotonic Dystrophy Type 1 (DM1). The program aims to address the underlying genetic cause of DM1 by targeting the *DMPK* (Dystrophia Myotonica Protein Kinase) gene to excise or disrupt the pathogenic CTG trinucleotide repeat expansions in the 3' untranslated region. These expansions lead to the accumulation of toxic RNA foci that sequester splicing proteins, resulting in multisystemic symptoms including muscle weakness, myotonia, and cardiac conduction defects. By utilizing CRISPR/Cas9 to reduce the burden of these toxic repeats, the therapy seeks to restore normal RNA processing and cellular function.

Other names

DMPK programCRISPR-Vertex DM1 program