Antisense oligonucleotides for the treatment of cardiomyopathy in Duchenne muscular dystrophy.

@article{Nguyen2019AntisenseOF,
  title={Antisense oligonucleotides for the treatment of cardiomyopathy in Duchenne muscular dystrophy.},
  author={Quynh Nguyen and Toshifumi Yokota},
  journal={American journal of translational research},
  year={2019},
  volume={11 3},
  pages={
          1202-1218
        }
}
Duchenne muscular dystrophy (DMD) is an X-linked recessive fatal neuromuscular disorder characterized by progressive muscle degeneration which affects one in 3500-5000 males born worldwide. DMD is caused by loss-of-function mutations in the dystrophin (DMD) gene encoding for dystrophin, a cytoskeletal protein that supports the structural integrity of myofibers during cycles of muscle contraction and relaxation. DMD patients do not only experience skeletal muscle deterioration but also severe… CONTINUE READING
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