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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Authors
Florian Eichler,Christine Duncan
Patricia Musolino,Paul Orchard,Satiro Oliveira,Adrian Thrasher,Myriam Armant,Colleen Dansereau,Troy Lund,Weston Miller,Gerald Raymond,Raman Sankar,Ami Shah,Caroline Sevin,H. Gaspar,Paul Gissen,Hernán Amartino,Drago Bratkovic,Nicholas Smith,Asif Paker,Esther Shamir,Tara O’Meara,David Davidson,Patrick Aubourg
+22 authors
,David WilliamsPublished
Oct 4, 2017
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Abstract
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 allogeneic hematopoietic stem-cell transplantation.
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