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Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy
by
Lauer, Arne
,
Speroni, Samantha L.
,
Choi, Myoung
,
Da, Xiao
,
Duncan, Christine
,
McCarthy, Siobhan
,
Krishnan, Vijai
,
Lusk, Cole A.
,
Rohde, David
,
Hansen, Mikkel Bo
,
Kalpathy-Cramer, Jayashree
,
Loes, Daniel J.
,
Caruso, Paul A.
,
Williams, David A.
,
Mouridsen, Kim
,
Emblem, Kyrre E.
,
Eichler, Florian S.
,
Musolino, Patricia L.
Published in
Nature communications
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692/617/375/1411
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Adrenoleukodystrophy
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Adrenoleukodystrophy - Genetics
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Autografts
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Blood-Brain Barrier
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Gene Dosage
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Gene Therapy
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