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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
(Massachusetts Medical Society, 2017-10-26)
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, ...