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

@article{Cartier2010HematopoieticSC,
  title={Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X‐Linked Adrenoleukodystrophy},
  author={Nathalie Cartier and Patrick R. Aubourg},
  journal={Brain Pathology},
  year={2010},
  volume={20}
}
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only therapeutic approach that can arrest cerebral demyelination of X‐linked adrenoleukodystrophy (ALD) in boys and results in long‐term in a good quality of life, provided the procedure is performed at an early stage of disease. Similar benefits of allogeneic HSCT have been demonstrated in adults with cerebral ALD. However, it is not yet known whether allogeneic HSCT can prevent or rescue adrenomyeloneuropathy. Allogeneic HSCT… 

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References

SHOWING 1-10 OF 57 REFERENCES

Treatment of X‐linked childhood cerebral adrenoleukodystrophy by the use of an allogeneic stem cell transplantation with reduced intensity conditioning regimen

Non‐myeloablative HSCT should be considered as an early treatment for X‐ALD, a rapidly progressive demyelinating condition affecting the cerebral white matter, which rapidly leads to total disability and death.

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

Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.

Matched Unrelated Human Umbilical Cord Blood Transplantation for X‐Linked Adrenoleukodystrophy

Unrelated cord blood transplantation may provide a good source of stem cells for patients with ALD when a matched sibling is not available.

Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.

  • D. BeamM. Poe M. Escolar
  • Medicine, Biology
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • 2007

Haematopoietic stem cell transplantation in 12 patients with cerebral X-linked adrenoleukodystrophy

It is confirmed that haematopoietic stem cell transplantation can stop the progress of demyelination when performed at a critical early stage of the disease.

Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999.

It is concluded that boys with early-stage disease benefit from HCT, whereas boys with advanced disease may be candidates for experimental therapies.

Successful allogeneic unrelated bone marrow transplantation using reduced‐intensity conditioning for the treatment of X‐linked adrenoleukodystrophy in a one‐yr‐old boy

A one‐yr‐old boy with ALD who received HLA‐matched unrelated BMT in an early stage of the disease after careful planning and observation since his birth has been shown that BMT should be considered when a child has a biochemical diagnosis and MRI findings of ALD without any neurological signs.

Unrelated Allogeneic Bone Marrow Transplant in Adrenoleukodystrophy Using CD34+ Stem Cell Selection

A case where the authors used CD34+ stem cell selection for allogeneic unrelated-donor BMT in a patient with adrenoleukodystrophy is reported here.
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