Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders

@article{Koppanati2009SystemicDO,
  title={Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders},
  author={Bhanu Munil Koppanati and Juan Li and Xiao Xiao and Paula R. Clemens},
  journal={Gene Therapy},
  year={2009},
  volume={16},
  pages={1130-1137}
}
One of the major challenges in the treatment of primary muscle disorders, which often affect many muscle groups, is achieving efficient, widespread transgene expression in muscle. In utero gene transfer can potentially address this problem by accomplishing the gene delivery when the tissue mass is small and the immune system is immature. Earlier studies with systemic in utero adeno-associated viral (AAV) vector serotype 1 gene delivery to embryonic day 16 (E-16) pups resulted in high levels of… CONTINUE READING
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