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| Verfasst von: | Lauer, Arne [VerfasserIn]  |
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| | Speroni, Samantha L. [VerfasserIn] |
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| | Choi, Myoung [VerfasserIn] |
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| | Da, Xiao [VerfasserIn] |
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| | Duncan, Christine [VerfasserIn] |
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| | McCarthy, Siobhan [VerfasserIn] |
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| | Krishnan, Vijai [VerfasserIn] |
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| | Lusk, Cole A. [VerfasserIn] |
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| | Rohde, David [VerfasserIn] |
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| | Hansen, Mikkel Bo [VerfasserIn] |
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| | Kalpathy-Cramer, Jayashree [VerfasserIn] |
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| | Loes, Daniel J. [VerfasserIn] |
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| | Caruso, Paul A. [VerfasserIn] |
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| | Williams, David A. [VerfasserIn] |
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| | Mouridsen, Kim [VerfasserIn] |
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| | Emblem, Kyrre E. [VerfasserIn] |
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| | Eichler, Florian S. [VerfasserIn] |
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| | Musolino, Patricia L. [VerfasserIn] |
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| Titel: | Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy |
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| Verf.angabe: | 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 |
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| E-Jahr: | 2023 |
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| Jahr: | 05 April 2023 |
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| Umfang: | 10 S. |
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| Fussnoten: | Gesehen am 23.06.2023 |
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| Titel Quelle: | Enthalten in: Nature Communications |
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| Ort Quelle: | [London] : Nature Publishing Group UK, 2010 |
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| Jahr Quelle: | 2023 |
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| Band/Heft Quelle: | 14(2023) vom: Apr., Artikel-ID 1900, Seite 1-10 |
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| ISSN Quelle: | 2041-1723 |
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| Abstract: | Blood-brain barrier disruption marks the onset of cerebral adrenoleukodystrophy (CALD), a devastating cerebral demyelinating disease caused by loss of ABCD1 gene function. The underlying mechanism are not well understood, but evidence suggests that microvascular dysfunction is involved. We analyzed cerebral perfusion imaging in boys with CALD treated with autologous hematopoietic stem-cells transduced with the Lenti-D lentiviral vector that contains ABCD1 cDNA as part of a single group, open-label phase 2-3 safety and efficacy study (NCT01896102) and patients treated with allogeneic hematopoietic stem cell transplantation. We found widespread and sustained normalization of white matter permeability and microvascular flow. We demonstrate that ABCD1 functional bone marrow-derived cells can engraft in the cerebral vascular and perivascular space. Inverse correlation between gene dosage and lesion growth suggests that corrected cells contribute long-term to remodeling of brain microvascular function. Further studies are needed to explore the longevity of these effects. |
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| DOI: | doi:10.1038/s41467-023-37262-w |
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| URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
kostenfrei: Volltext: https://doi.org/10.1038/s41467-023-37262-w |
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| | kostenfrei: Volltext: https://www.nature.com/articles/s41467-023-37262-w |
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| | DOI: https://doi.org/10.1038/s41467-023-37262-w |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| Sach-SW: | Demyelinating diseases |
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| | Neurodegeneration |
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| K10plus-PPN: | 1850879524 |
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| Verknüpfungen: | → Zeitschrift |
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Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy / Lauer, Arne [VerfasserIn]; 05 April 2023 (Online-Ressource)
