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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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Detalles Bibliográficos
Publicado en:Brain Pathol
Autores principales: Cartier, Nathalie, Aubourg, Patrick
Formato: Artigo
Lenguaje:Inglês
Publicado: Blackwell Publishing Ltd 2010
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Acceso en línea: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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