Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

@article{Cartier2009HematopoieticSC,
  title={Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy},
  author={N. Cartier and S. Hacein-Bey-Abina and C. Bartholomae and G. Veres and Manfred Schmidt and Ina Kutschera and M. Vidaud and U. Abel and L. Dal-Cortivo and L. Caccavelli and N. Mahlaoui and V. Kiermer and D. Mittelstaedt and C. Bellesme and N. Lahlou and F. Lefr{\`e}re and S. Blanche and M. Audit and E. Payen and P. Leboulch and Bruno L'homme and P. Bougn{\`e}res and C. von Kalle and A. Fischer and M. Cavazzana‐Calvo and P. Aubourg},
  journal={Science},
  year={2009},
  volume={326},
  pages={818 - 823}
}
Slowing Brain Disease with Gene Therapy X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie “Lorenzo's Oil,” is typically treated by transplantation of bone marrow from matched donors. This treatment slows progression of the disease by introducing cells that differentiate into myelin-producing cells. Cartier et al. (p. 818; see Perspective by Naldini) tested an alternative gene therapy–based approach in two young patients without… Expand
[Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector].
TLDR
The results suggest that lentiviral vectors are suitable for transferring therapeutic genes to hematopoietic stem cells, and provide the first example of successful gene therapy for a severe neurodegenerative disease. Expand
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The reconstitution of ARSA activity in the cerebrospinal fluid and the arrested progression of neurodegenerative disease in the three treated patients demonstrate that the transplanted cells, or their progeny, can seed the nervous system and deliver therapeutic levels of active enzyme. Expand
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Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X‐Linked Adrenoleukodystrophy
TLDR
Recently, HSC gene therapy using lentiviral vector was shown to have comparable efficacy than allogeneic HSCT in two boys with cerebral ALD who had no Human‐leukocyte‐antigen (HLA)‐matched donor and may become the first therapeutic option for all ALD male patients who develop cerebral demyelination. Expand
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