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. |
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| 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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| ISSN: | 2073-4409 |
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| DOI: | 10.3390/cells14241942 |
