Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution.

@article{Ciron2009HumanAG,
  title={Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution.},
  author={C Ciron and Arnaud Cressant and Françoise Roux and Sylvie Raoul and Yan Ch{\'e}rel and Philippe Hantraye and Nicole D{\'e}glon and Bertrand Schwartz and Martine Barkats and J F Heard and Marc Tardieu and Philippe Moullier and M. C. De Colle},
  journal={Human gene therapy},
  year={2009},
  volume={20 4},
  pages={350-60}
}
We have previously demonstrated that delivery of a recombinant adeno-associated virus (rAAV) encoding human alpha-iduronidase (hIDUA) in the putamen and centrum semiovale was feasible and beneficial in a dog model of Hurler's syndrome. In the present study, we investigated the safety and vector diffusion profile of three rAAV serotypes (rAAV2/1, rAAV2/2, and rAAV2/5), encoding hIDUA in the central and peripheral nervous systems of nonhuman primates. Six macaques received the same vector dose… CONTINUE READING
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