Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

@article{Cressant2004ImprovedBA,
  title={Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.},
  author={Arnaud Cressant and Nathalie Desmaris and Lucie V{\'e}rot and Thomas Br{\'e}jot and Roselyne Froissart and Marie-T Vanier and Ir{\`e}ne Maire and Jean Michel Heard},
  journal={The Journal of neuroscience : the official journal of the Society for Neuroscience},
  year={2004},
  volume={24 45},
  pages={10229-39}
}
Sanfilippo syndrome is a mucopolysaccharidosis (MPS) caused by a lysosomal enzyme defect interrupting the degradation pathway of heparan sulfates. Affected children develop hyperactivity, aggressiveness, delayed development, and severe neuropathology. We observed relevant behaviors in the mouse model of Sanfilippo syndrome type B (MPSIIIB), in which the gene coding for alpha-N-acetylglucosaminidase (NaGlu) is invalidated. We addressed the feasibility of gene therapy in these animals. Vectors… CONTINUE READING
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