AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice.

  title={AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice.},
  author={Brian P Bostick and Jin-Hong Shin and Yongping Yue and Dongsheng Duan},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={19 10},
Dystrophin deficiency leads to lethal dilated Duchenne cardiomyopathy. A promising therapy is to deliver a highly abbreviated microdystrophin gene to the heart using adeno-associated virus (AAV). Microdystrophin has been shown to mitigate dystrophin-deficient skeletal muscle disease. However, it is not clear whether microdystrophin is equally effective in treating Duchenne cardiomyopathy. To evaluate microdystrophin therapy in the heart, we injected 5 × 10(12) viral genome particles/mouse of… CONTINUE READING


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