Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.

@article{Sun2005CorrectionOG,
  title={Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.},
  author={Baodong Sun and Haoyue Zhang and Luis M Franco and Talmage T. Brown and Andrew Bird and Ayn Schneider and Dwight D. Koeberl},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2005},
  volume={11 6},
  pages={889-98}
}
Glycogen storage disease type II (Pompe disease) causes death in infancy from cardiorespiratory failure due to acid alpha-glucosidase (GAA; acid maltase) deficiency. An AAV2 vector pseudotyped as AAV6 (AAV2/6 vector) transiently expressed high-level human GAA in GAA-knockout (GAA-KO) mice without reducing glycogen storage; however, in immunodeficient GAA-KO/SCID mice the AAV2/6 vector expressed high-level GAA and reduced the glycogen content of the injected muscle for 24 weeks. A CD4+/CD8… CONTINUE READING

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