Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@article{Denti2006BodywideGT,
  title={Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.},
  author={Michela Denti and Alessandro Rosa and Giuseppe D'Antona and Olga Sthandier and Fernanda Gabriella De Angelis and Carmine Nicoletti and Mariacarmela Allocca and Orietta Pansarasa and Valeria Parente and Antonio Musar{\`o} and Alberto Auricchio and Roberto Bottinelli and Irene Bozzoni},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={2006},
  volume={103 10},
  pages={
          3758-63
        }
}
Duchenne muscular dystrophy is an X-linked muscle disease characterized by mutations in the dystrophin gene. Many of these can be corrected at the posttranscriptional level by skipping the mutated exon. We have obtained persistent exon skipping in mdx mice by tail vein injection with an adeno-associated viral (AAV) vector expressing antisense sequences as part of the stable cellular U1 small nuclear RNA. Systemic delivery of the AAV construct resulted in effective body-wide colonization… CONTINUE READING

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Muscle Gene Therapy

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Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.

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  • 2008
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Genome Editing in Neurosciences

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