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 G. Danialou and N. Larochelle and M. Bourget and Joon-shik Moon and A. Liu and R. Gilbert and B. Petrof and J. Nalbantoglu and G. Karpati},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2007},
  volume={15 10},
  pages={
          1767-74
        }
}
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… Expand
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