Duchenne Cardiomyopathy Gene Therapy

  • Jin-Hong Shin
  • Brian Bostick
  • Deborah M. Fine
  • Yongping Yue
  • Dongsheng Duan


Duchenne cardiomyopathy is a heart disease resulting from the loss of cardiac dystrophin. It significantly reduces the life quality and shortens lifespan in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and X-linked dilated cardiomyopathy patients and carriers. Gene replacement therapy with adeno-associated viral vector (AAV) and gene repair therapy with exon skipping hold great promise for restoring dystrophin expression and ameliorating cardiomyopathy. The last few years have witnessed tremendous advances towards Duchenne cardiomyopathy gene therapy. The infrastructure (animal models and functional assays) is now available for comprehensive preclinical studies. Essential parameters, such as the therapeutic threshold, have also been defined. Together with the recent developments in novel AAV vectors and modified antisense oligonucleotides, clinical application of Duchenne cardiomyopathy gene therapy may become a reality in the near future.


Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy Patient Dystrophin Gene Becker Muscular Dystrophy 



Research on Duchenne muscular dystrophy in Duan lab is supported by grants from the National Institutes of Health AR-49419, AR-57209 and NS-62934 (DD) and the Muscular Dystrophy Association (DD).


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Copyright information

© Springer Science+Business Media, LLC 2010

Authors and Affiliations

  • Jin-Hong Shin
  • Brian Bostick
  • Deborah M. Fine
  • Yongping Yue
  • Dongsheng Duan
    • 1
  1. 1.Department of Molecular Microbiology and ImmunologySchool of Medicine, The University of Missouri, One Hospital Dr.ColumbiaUSA

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