DMD Gene Repair Strategy Takes a Big Step Forward

Researchers report making a 10-fold improvement in a technology that permanently repairs flawed dystrophin genes, giving a real boost to this therapeutic strategy.


  • An MDA-supported research group at the University of California, Los Angeles, has repaired the flawed dystrophin gene in isolated muscle cells and in mdx mice, an experimental animal model of Duchenne muscular dystrophy.
  • Newly published refinements in the technology demonstrate a 10-fold improvement in the efficiency of gene repair.
  • Although further refinements are needed, the results represent a "proof of concept" for gene repair as a permanent therapy for DMD.

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I love to see this.
Wish I understoon what further refinements were needed and an estimated timeframe fur such. This is really great news Melanie!! So glad you found it, I've all but given up on gene therapy.

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