AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease.

@article{Passini2005AAVVC,
  title={AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease.},
  author={Marco A. Passini and Shannon L Macauley and Michael R. Huff and Tatyana V. Taksir and Jie Bu and I-Huan Wu and Peter A. Piepenhagen and J. C. Dodge and Lamya S. Shihabuddin and Catherine R. O’Riordan and Edward H Schuchman and Gregory R. Stewart},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2005},
  volume={11 5},
  pages={
          754-62
        }
}
  • Marco A. Passini, Shannon L Macauley, +9 authors Gregory R. Stewart
  • Published in
    Molecular therapy : the…
    2005
  • Biology, Medicine
  • Niemann-Pick A disease (NPA) is a fatal lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. The lack of functional ASM results in cellular accumulation of sphingomyelin and cholesterol within distended lysosomes throughout the brain. In this study, we investigated the potential of AAV-mediated expression of ASM to correct the brain pathology in an ASM knockout (ASMKO) mouse model of NPA. An AAV serotype 2 vector encoding human ASM (AAV2-hASM) was injected… CONTINUE READING

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