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    • Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. 

      Eichler, Florian.; Duncan, Christine.; Musolino, Patricia.; Orchard, Paul.; Oliveira, Satiro.; Thrasher, Adrian.; Amartino, Hernán.; et al. (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, ...