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

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. Simil...

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Dades bibliogràfiques
Publicat a:Brain Pathol
Autors principals: Cartier, Nathalie, Aubourg, Patrick
Format: Artigo
Idioma:Inglês
Publicat: Blackwell Publishing Ltd 2010
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8094635/
https://ncbi.nlm.nih.gov/pubmed/20626747
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/j.1750-3639.2010.00394.x
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