Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy.

@article{Lin2007CentralNS,
  title={Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy.},
  author={Darshong Lin and Anthony Donsante and Shannon Macauley and Beth Levy and Carole Vogler and Mark S Sands},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2007},
  volume={15 1},
  pages={44-52}
}
Globoid-cell leukodystrophy (GLD) is a rapidly progressing inherited neurodegenerative disorder caused by a deficiency in galactosylceramidase activity. Previous studies in the murine model of GLD (Twitcher mouse) have shown that both bone marrow transplantation (BMT) and central nervous system (CNS)-directed gene therapy can be moderately effective at ameliorating certain aspects of GLD. As BMT and CNS-directed gene therapy target fundamentally different tissues, we tested the hypothesis that… CONTINUE READING

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