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Durable Global Correction of CNS and PNS and Lifespan Rescue in Murine Globoid Cell Leukodystrophy via AAV9-Mediated Monotherapy.

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Title: Durable Global Correction of CNS and PNS and Lifespan Rescue in Murine Globoid Cell Leukodystrophy via AAV9-Mediated Monotherapy.
Authors: Lin DS; Department of Translational Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.; Department of Medicine, MacKay Medical University, New Taipei 252005, Taiwan.; Department of Pediatrics, MacKay Memorial Hospital, Taipei 104217, Taiwan., Ho CS; Department of Medicine, MacKay Medical University, New Taipei 252005, Taiwan.; Department of Neurology, MacKay Children's Hospital, Taipei 104217, Taiwan., Huang YW; Department of Medical Research, MacKay Memorial Hospital, Taipei 104217, Taiwan., Lee TH; Department of Medical Research, MacKay Memorial Hospital, Taipei 104217, Taiwan., Huang ZD; Department of Medical Research, MacKay Memorial Hospital, Taipei 104217, Taiwan., Wang TJ; Department of Laboratory Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan., Cheng WC; Department of Laboratory Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.; Department of Laboratory Medicine, School of Medicine, College of Medicine, National Taiwan University, Taipei 106319, Taiwan., Huang SF; Department of Laboratory Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.
Source: Cells [Cells] 2025 Dec 08; Vol. 14 (24). Date of Electronic Publication: 2025 Dec 08.
Publication Type: Journal Article
Journal Info: Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409 (Electronic) Linking ISSN: 20734409 NLM ISO Abbreviation: Cells Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Durable Global Correction of CNS and PNS and Lifespan Rescue in Murine Globoid Cell Leukodystrophy via AAV9-Mediated Monotherapy.
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  Data: <searchLink fieldCode="AU" term="%22Lin+DS%22">Lin DS</searchLink>; Department of Translational Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.; Department of Medicine, MacKay Medical University, New Taipei 252005, Taiwan.; Department of Pediatrics, MacKay Memorial Hospital, Taipei 104217, Taiwan.<br /><searchLink fieldCode="AU" term="%22Ho+CS%22">Ho CS</searchLink>; Department of Medicine, MacKay Medical University, New Taipei 252005, Taiwan.; Department of Neurology, MacKay Children's Hospital, Taipei 104217, Taiwan.<br /><searchLink fieldCode="AU" term="%22Huang+YW%22">Huang YW</searchLink>; Department of Medical Research, MacKay Memorial Hospital, Taipei 104217, Taiwan.<br /><searchLink fieldCode="AU" term="%22Lee+TH%22">Lee TH</searchLink>; Department of Medical Research, MacKay Memorial Hospital, Taipei 104217, Taiwan.<br /><searchLink fieldCode="AU" term="%22Huang+ZD%22">Huang ZD</searchLink>; Department of Medical Research, MacKay Memorial Hospital, Taipei 104217, Taiwan.<br /><searchLink fieldCode="AU" term="%22Wang+TJ%22">Wang TJ</searchLink>; Department of Laboratory Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.<br /><searchLink fieldCode="AU" term="%22Cheng+WC%22">Cheng WC</searchLink>; Department of Laboratory Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.; Department of Laboratory Medicine, School of Medicine, College of Medicine, National Taiwan University, Taipei 106319, Taiwan.<br /><searchLink fieldCode="AU" term="%22Huang+SF%22">Huang SF</searchLink>; Department of Laboratory Medicine, MacKay Memorial Hospital, Taipei 104217, Taiwan.
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  Data: <searchLink fieldCode="JN" term="%22101600052%22">Cells</searchLink> [Cells] 2025 Dec 08; Vol. 14 (24). <i>Date of Electronic Publication: </i>2025 Dec 08.
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        Value: 10.3390/cells14241942
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        Text: English
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      – TitleFull: Durable Global Correction of CNS and PNS and Lifespan Rescue in Murine Globoid Cell Leukodystrophy via AAV9-Mediated Monotherapy.
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              Text: 2025 Dec 08
              Type: published
              Y: 2025
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