Nature Communications (Apr 2023)
Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy
- Arne Lauer,
- Samantha L. Speroni,
- Myoung Choi,
- Xiao Da,
- Christine Duncan,
- Siobhan McCarthy,
- Vijai Krishnan,
- Cole A. Lusk,
- David Rohde,
- Mikkel Bo Hansen,
- Jayashree Kalpathy-Cramer,
- Daniel J. Loes,
- Paul A. Caruso,
- David A. Williams,
- Kim Mouridsen,
- Kyrre E. Emblem,
- Florian S. Eichler,
- Patricia L. Musolino
Affiliations
- Arne Lauer
- Department of Neurology, Massachusetts General Hospital
- Samantha L. Speroni
- Department of Neurology, Massachusetts General Hospital
- Myoung Choi
- Department of Neurology, Massachusetts General Hospital
- Xiao Da
- Functional Neuroimaging Laboratory, Department of Psychiatry, Brigham and Women’s Hospital
- Christine Duncan
- Dana–Farber and Boston Children’s Cancer and Blood Disorders Center and Harvard Medical School
- Siobhan McCarthy
- Department of Neurology, Massachusetts General Hospital
- Vijai Krishnan
- Department of Neurology, Massachusetts General Hospital
- Cole A. Lusk
- Department of Neurology, Massachusetts General Hospital
- David Rohde
- Center for Systems Biology, Massachusetts General Hospital
- Mikkel Bo Hansen
- Department of Clinical Medicine, Aarhus University
- Jayashree Kalpathy-Cramer
- Athinoula A. Martinos Centre for Biomedical Imaging
- Daniel J. Loes
- Suburban Radiologic Consultants, Ltd
- Paul A. Caruso
- Department of Radiology, Massachusetts General Hospital
- David A. Williams
- Dana–Farber and Boston Children’s Cancer and Blood Disorders Center and Harvard Medical School
- Kim Mouridsen
- Department of Clinical Medicine, Aarhus University
- Kyrre E. Emblem
- Department of Diagnostic Physics, Oslo University Hospital
- Florian S. Eichler
- Department of Neurology, Massachusetts General Hospital
- Patricia L. Musolino
- Department of Neurology, Massachusetts General Hospital
- DOI
- https://doi.org/10.1038/s41467-023-37262-w
- Journal volume & issue
-
Vol. 14,
no. 1
pp. 1 – 10
Abstract
Cerebral adrenoleukodystrophy (CALD) is a demyelinating disease caused by loss of ABCD1 gene function. Here the authors investigate white matter structural and microvascular changes in boys with CALD that received gene therapy with autologous hematopoietic stem-cells.
