Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II.
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| Title: | Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II. |
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| Authors: | Pyne NK; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43215, USA., Bagel J; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Shyng C; Gene Therapy Center, University of North Carolina - Chapel Hill, Chapel Hill, NC 27599, USA., Odonnell P; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Miyadera K; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Srnak J; M6P Therapeutics, St. Louis, MO 63108, USA., Swain G; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Pesayco JP; Department of Pathology, School of Medicine, University of California, San Diego, La Jolla, CA 92093, USA., Shelton GD; Department of Pathology, School of Medicine, University of California, San Diego, La Jolla, CA 92093, USA., Assenmacher CA; Comparative Pathology Core, Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA., Dickson P; Department of Pediatrics, Washington University, St. Louis, MO 63110, USA., Stern JA; Department of Clinical Sciences, College of Veterinary Medicine, North Carolina State University, Raleigh, NC 27607, USA., Flanagan-Steet H; JC Self Research Institute, Greenwood Genetic Center, Greenwood, SC 29646, USA., Gray SJ; Gene Therapy Center, University of North Carolina - Chapel Hill, Chapel Hill, NC 27599, USA., Bradbury AM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43215, USA. Electronic address: allison.bradbury@nationwidechildrens.org. |
| Source: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Aug 06; Vol. 33 (8), pp. 3808-3821. Date of Electronic Publication: 2025 Apr 24. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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