Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

@article{Peccate2016AntisensePI,
  title={Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.},
  author={C{\'e}cile Peccate and Am{\'e}d{\'e}e Mollard and Ma{\"e}va le Hir and Laura Julien and Graham McClorey and Susan A Jarmin and Anita Le Heron and George R. Dickson and Sofia Benkhelifa-Ziyyat and France Pi{\'e}tri-Rouxel and Matthew J. Wood and Thomas Voit and St{\'e}phanie Lorain},
  journal={Human molecular genetics},
  year={2016},
  volume={25 16},
  pages={
          3555-3563
        }
}
In preclinical models for Duchenne muscular dystrophy, dystrophin restoration during adeno-associated virus (AAV)-U7-mediated exon-skipping therapy was shown to decrease drastically after six months in treated muscles. This decline in efficacy is strongly correlated with the loss of the therapeutic AAV genomes, probably due to alterations of the dystrophic myofiber membranes. To improve the membrane integrity of the dystrophic myofibers at the time of AAV-U7 injection, mdx muscles were pre… CONTINUE READING
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