Gene-Editing Therapies for the Treatment of Duchenne Muscular Dystrophy (DMD)

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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 a functional form of dystrophin protein.

Gene-Editing Therapies for the Treatment of Duchenne Muscular Dystrophy (DMD) is protected by three U.S. patents, as well as patents in Canada, Australia, and Mexico (with additional patents pending in the U.S. and in other countries). Of note, certain granted patents include claims that are not limited by specific target sequence, mRNA sequence or chemistry, cell type, or method of transfection.

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

Representative Data

Figure 1. Gene editing of the DMD gene in primary human epidermal keratinocytes.

Representative Claim

U.S. Pat. No. 10,752,918

An in vitro method for producing a gene-edited cell comprising transfecting in vitro a cell comprising a Duchenne muscular dystrophy (DMD) gene with a nucleic acid encoding a gene-editing protein, wherein the gene-editing protein comprises: (a) a DNA-binding domain and (b) a nuclease domain and is capable of binding to the DMD gene, wherein:

(a) the DNA-binding domain comprises a plurality of repeat sequences and at least one of the repeat sequences comprises the amino acid sequence: LTPvQWAlAwxyzGHGG (SEQ ID NO: 75) and is between 36 and 39 amino acids long, wherein:

“v” is Q, D or E,

“w” is S or N,

“x” is N or H,

“y” is D, A, H, N, K, or G, and


(b) the nuclease domain comprises a catalytic domain of a nuclease, to result in a gene-edited cell.