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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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Dettagli Bibliografici
Pubblicato in:Brain Pathol
Autori principali: Cartier, Nathalie, Aubourg, Patrick
Natura: Artigo
Lingua:Inglês
Pubblicazione: Blackwell Publishing Ltd 2010
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Accesso online: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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