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Article

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

4 Oct 2017

Abstract excerpt

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 allogeneic hematopoietic stem-cell transplantation. METHODS: We enrolled boys with cerebral adrenoleukodystrophy in a...

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PMID: 28976817
DOI: 10.1056/nejmoa1700554

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