Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy

HIGHLIGHTS

  • who: Arne Lauer from the (UNIVERSITY) have published the research work: Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy, in the Journal: (JOURNAL) of 25/09/2020
  • what: The authors demonstrate that ABCD1 functional bone marrow-derived cells can engraft in the cerebral vascular and perivascular space. The authors show that GT leads to widespread and sustained normalization of white matter permeability and microvascular flow. The authors propose that improved microvascular flow reflects normalized interactions between corrected circulating leukocytes and endothelial cells, which attenuates the progression of . . .

     

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