Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease.

@article{Galbiati2009CombinedHA,
  title={Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease.},
  author={F. Galbiati and Maria Irene Givogri and Ludovico Cantuti and Alejandro L Rosas and Hao-qiang Cao and Richard van Breemen and Ernesto R Bongarzone},
  journal={Journal of neuroscience research},
  year={2009},
  volume={87 8},
  pages={1748-59}
}
This study characterized the therapeutic benefits of combining hematogenous cell replacement with lentiviral-mediated gene transfer of galactosylceramidase (GALC) in Twitcher mice, a bona fide model for Krabbe disease. Bone marrow cells and GALC-lentiviral vectors were administered intravenously without any preconditioning to newborn Twitcher pups before postnatal day 2. Treated Twitchers survived up to 4 months of age. GALC activity remained less than 5% of normal values in the nervous system… CONTINUE READING

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