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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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Bibliografiske detaljer
Udgivet i:Brain Pathol
Main Authors: Cartier, Nathalie, Aubourg, Patrick
Format: Artigo
Sprog:Inglês
Udgivet: Blackwell Publishing Ltd 2010
Fag:
Online adgang: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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