Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy.
Lauer A, Speroni SL, Choi M, Da X, Duncan C, McCarthy S, Krishnan V, Lusk CA, Rohde D, Hansen MB, Kalpathy-Cramer J, Loes DJ, Caruso PA, Williams DA, Mouridsen K, Emblem KE, Eichler FS, Musolino PL.
Lauer A, et al. Among authors: emblem ke.
Nat Commun. 2023 Apr 5;14(1):1900. doi: 10.1038/s41467-023-37262-w.
Nat Commun. 2023.
PMID: 37019892
Free PMC article.