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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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Detalhes bibliográficos
Publicado no:Brain Pathol
Main Authors: Cartier, Nathalie, Aubourg, Patrick
Formato: Artigo
Idioma:Inglês
Publicado em: Blackwell Publishing Ltd 2010
Assuntos:
Acesso em linha: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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