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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, ...
Hematopoietic Cell Transplantation-Specific Comorbidity Index Predicts Morbidity and Mortality in Autologous Stem Cell Transplantation.
(Elsevier, 2017-10-01)
The hematopoietic cell transplantation-specific comorbidity index (HCT-CI) score is a useful tool to assess the risk for nonrelapse mortality (NRM) after allogeneic hematopoietic stem cell transplantation. Although the ...