Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

BACKGROUND: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allo...

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Gepubliceerd in:N Engl J Med
Hoofdauteurs: Eichler, Florian, Duncan, Christine, Musolino, Patricia L., Orchard, Paul J., De Oliveira, Satiro, Thrasher, Adrian J., Armant, Myriam, Dansereau, Colleen, Lund, Troy C., Miller, Weston P., Raymond, Gerald V., Sankar, Raman, Shah, Ami J., Sevin, Caroline, Gaspar, H. Bobby, Gissen, Paul, Amartino, Hernan, Bratkovic, Drago, Smith, Nicholas J.C., Paker, Asif M., Shamir, Esther, O’Meara, Tara, Davidson, David, Aubourg, Patrick, Williams, David A.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2017
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5708849/
https://ncbi.nlm.nih.gov/pubmed/28976817
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1700554
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