Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice

@article{Fu2007SignificantlyIL,
  title={Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice},
  author={Haiyan Fu and Lu Kang and Jennifer S Jennings and Sheryl S. Moy and Adryan Perez and Julianne D Dirosario and Douglas M Mccarty and Joseph Muenzer},
  journal={Gene Therapy},
  year={2007},
  volume={14},
  pages={1065-1077}
}
Mucopolysaccharidosis (MPS) IIIB is an inherited lysosomal storage disease, caused by the deficiency of α-N-acetylglucosaminidase (NaGlu), resulting in severe global neurological involvement with high mortality. One major hurdle in therapeutic development for MPS IIIB is the presence of the blood–brain barrier, which impedes the global central nervous system (CNS) delivery of therapeutic materials. In this study, we used a minimal invasive strategy, combining an intravenous (i.v.) and an… CONTINUE READING
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Techniques in Diagnostic Human Biochemical Genetics – A Laboratory Manual

  • Wiley-Liss: New York,
  • 1991
Highly Influential
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