Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.

@article{Fraldi2007FunctionalCO,
  title={Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.},
  author={Alessandro Fraldi and Kim M Hemsley and Allison C Crawley and Alessia Lombardi and Adeline A Lau and Leanne Sutherland and Alberto Auricchio and Andrea Ballabio and John J. Hopwood},
  journal={Human molecular genetics},
  year={2007},
  volume={16 22},
  pages={2693-702}
}
Mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome) is a lysosomal storage disorder caused by the congenital deficiency of sulfamidase (SGSH) enzyme and consequent accumulation of partially degraded heparan sulfate (HS) in lysosomes. The central nervous system (CNS) is the predominant site of tissue damage in MPS-IIIA. Here we describe a gene therapy approach for MPS-IIIA in a mouse model using recombinant adeno-associated virus serotype 5 (AAV2/5) as a vehicle to deliver… CONTINUE READING
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