Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

@inproceedings{Fu2016FunctionalCO,
  title={Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery},
  author={Haiyan Fu and Marcela P Cataldi and Tierra Amber Ware and Kimberly Zaraspe and Aaron S. Meadows and Darren A Murrey and Douglas M Mccarty},
  booktitle={Molecular therapy. Methods & clinical development},
  year={2016}
}
The reversibility of neuropathic lysosomal storage diseases, including MPS IIIA, is a major goal in therapeutic development, due to typically late diagnoses and a large population of untreated patients. We used self-complementary adeno-associated virus (scAAV) serotype 9 vector expressing human N-sulfoglucosamine sulfohydrolase (SGSH) to test the efficacy of treatment at later stages of the disease. We treated MPS IIIA mice at 1, 2, 3, 6, and 9 months of age with an intravenous injection of… CONTINUE READING
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