Gene therapy for leukodystrophies.

@article{Biffi2011GeneTF,
  title={Gene therapy for leukodystrophies.},
  author={Alessandra Biffi and Patrick R. Aubourg and Nathalie Cartier},
  journal={Human molecular genetics},
  year={2011},
  volume={20 R1},
  pages={
          R42-53
        }
}
Leukodystrophies (LDs) refer to a group on inherited diseases in which molecular abnormalities of glial cells are responsible for exclusive or predominant defects in myelin formation and/or maintenance within the central and, sometimes, the peripheral nervous system. For three of them [X-linked adrenoleukodystrophy (X-ALD), metachromatic (MLD) and globoid cell LDs], a gene therapy strategy aiming at transferring the disease gene into autologous hematopoietic stem cells (HSCs) using lentiviral… 

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...

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