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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Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

It is demonstrated here that neonatal lentiviral vector-mediated intracerebral gene therapy (IC GT) or transplantation of GALC-overexpressing neural stem cells (NSC) synergize with bone marrow transplant (BMT) providing dramatic extension of lifespan and global clinical–pathological rescue in a relevant GLD murine model.

Gene-based approaches to inherited neurometabolic diseases.

An overview of the gene therapy strategies currently under clinical investigation for neurometabolic Lysosomal and Peroxisomal Storage Diseases such as Adreno and Metachromatic Leukodystrophies, as well as novel emerging indications as Mucopolysaccharidoses, Gangliosidoses and Neuronal Ceroid Lipofuscinoses are provided.
...

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