Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Volume: 377, Issue: 17, Pages: 1630 - 1638
Published: Oct 26, 2017
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...
Paper Details
Title
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Published Date
Oct 26, 2017
Volume
377
Issue
17
Pages
1630 - 1638
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