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Gene-Editing Therapies for Duchenne Muscular Dystrophy (DMD)


Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, which encodes dystrophin, a protein normally expressed in skeletal muscle.

Our scientists developed a method for treating DMD by using gene-editing proteins to edit the DMD gene to result in the production of an active form of dystrophin protein.

Gene-Editing Therapies for Duchenne Muscular Dystrophy (DMD) is protected by three pending U.S. patents (with additional patents pending in other countries).

Example Applications

  • Alter dystrophin mRNA splicing, e.g., ablate the splice acceptor site upstream of a mutation-containing exon to generate functional dystrophin protein
  • Deliver the therapy directly to the patient’s skeletal muscle – avoid ex vivo cell manipulation
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to various cells and tissues ex vivo and in vivo