Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.

@article{Wolf2011DirectGT,
  title={Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.},
  author={Daniel A. Wolf and Andrew W. Lenander and Zhenhong Nan and Lalitha R. Belur and Chester B. Whitley and Pankaj Gupta and Walter C. Low and R. Scott McIvor},
  journal={Neurobiology of disease},
  year={2011},
  volume={43 1},
  pages={123-33}
}
The mucopolysaccharidoses (MPSs) are a group of 11 storage diseases caused by disruptions in glycosaminoglycan (GAG) catabolism, leading to their accumulation in lysosomes. Resultant multisystemic disease is manifested by growth delay, hepatosplenomegaly, skeletal dysplasias, cardiopulmonary obstruction, and, in severe MPS I, II, III, and VII, progressive neurocognitive decline. Some MPSs are treated by allogeneic hematopoietic stem cell transplantation (HSCT) and/or recombinant enzyme… CONTINUE READING
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