Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems

@article{Mattar2013SystemicDO,
  title={Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems},
  author={Citra N. Z. Mattar and Simon N Waddington and Akm Abdul Ahad Biswas and Nuryanti B Johana and Xue Wen Ng and Angus S. Fisk and Nicholas M Fisk and L. G. Tan and Ahad A Rahim and Suzanne M. K. Buckley and Mei Hong Tan and Jie Lu and Mahesh Choolani and Jerry K. Y. Chan},
  journal={Gene Therapy},
  year={2013},
  volume={20},
  pages={69-83}
}
Correction of perinatally lethal neurogenetic diseases requires efficient transduction of several cell types within the relatively inaccessible CNS. Intravenous AAV9 delivery in mouse has achieved development stage-specific transduction of neuronal cell types, with superior neuron-targeting efficiency demonstrated in prenatal compared with postnatal recipients. Because of the clinical relevance of the non-human primate (NHP) model, we investigated the ability of AAV9 to transduce the NHP CNS… CONTINUE READING
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