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={Nathalie Cartier and Salima Hacein-Bey-Abina and Cynthia C. Bartholomae and Gabor Istvan Veres and Manfred Schmidt and Ina Kutschera and Michel Vidaud and Ulrich Abel and Liliane Dal-Cortivo and Laure Caccavelli and Nizar Mahlaoui and V{\'e}ronique Kiermer and Denice M. Mittelstaedt and C{\'e}line Bellesme and Najiba Lahlou and François Lefrère and St{\'e}phane Blanche and Muriel Audit and Emmanuel Payen and Philippe Leboulch and Bruno L'homme and Pierre Bougn{\`e}res and Christof von Kalle and Alain Fischer and Marina Cavazzana‐Calvo and Patrick R. 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… 

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Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X‐Linked Adrenoleukodystrophy

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.

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Major recent progress has been made in haemoglobinopathies, such as beta‐thalassaemia, sickle cell disease and Fanconi anaemia, and also specific lysosomal storage diseases, which, although not strictly bone marrow specific conditions, have been effectively treated by bone marrow‐based treatment.

Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy

A focused literature review was performed using the terms ‘hematopoietic stem cell transplantation,’ ‘gene therapy’ and ‘adrenoleukodystrophy’ to include relevant literature, especially comparing the experience with gene therapy and HSCT outcomes, to review the history and experience with HSCT in cerebral ALD.

Gene therapy for leukodystrophies.

A gene therapy strategy aiming at transferring the disease gene into autologous hematopoietic stem cells (HSCs) using lentiviral vectors has been developed and has already entered into the clinics for X-ALD and MLD.

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

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