Successful compensation for dystrophin deficiency by a helper-dependent adenovirus expressing full-length utrophin.

@article{Deol2007SuccessfulCF,
  title={Successful compensation for dystrophin deficiency by a helper-dependent adenovirus expressing full-length utrophin.},
  author={Jatinderpal R. Deol and Gawiyou Danialou and Nancy Larochelle and Myl{\`e}ne Bourget and Joon-shik Moon and An-Bang Liu and R{\'e}nald Gilbert and Basil J Petrof and Jos{\'e}phine Nalbantoglu and George Karpati},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2007},
  volume={15 10},
  pages={
          1767-74
        }
}
  • Jatinderpal R. Deol, Gawiyou Danialou, +7 authors George Karpati
  • Published in
    Molecular therapy : the…
    2007
  • Biology, Medicine
  • Helper-dependent adenovirus vector (AdV)-mediated full-length dystrophin expression leads to significant mitigation of the dystrophic phenotype of the mdx mouse. However, dystrophin, as a neoantigen, elicits antibody formation. As an alternative approach, we evaluated gene transfer of full-length murine utrophin, a functional homologue of dystrophin that is normally present only at the neuromuscular junction. A single injection in the tibialis anterior (TA) muscle of the helper-dependent… CONTINUE READING

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