Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.

@article{Benhamida2003TransducedCC,
  title={Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.},
  author={Sonia Benhamida and Francoise Pflumio and Anne Dubart-Kupperschmitt and Jing Chao Zhao-Emonet and Marina Cavazzana-Calvo and Francis Rocchiccioli and Serge Fichelson and Patrick R Aubourg and Pierre Charneau and Nathalie Cartier},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2003},
  volume={7 3},
  pages={317-24}
}
X-linked adrenoleukodystrophy (ALD), an inherited demyelinating disorder of the central nervous system, can be corrected by allogeneic bone marrow transplantation, likely due to the turnover of brain macrophages that are bone marrow derived. ALD is characterized by an accumulation of very long chain fatty acids (VLCFA) due to the deficiency of an ATP binding cassette transporter that imports these fatty acids in peroxisomes. Murine retroviral transduction results in metabolic correction of ALD… CONTINUE READING
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