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.
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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  Data: Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II.
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  Data: <searchLink fieldCode="AU" term="%22Pyne+NK%22">Pyne NK</searchLink>; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43215, USA.<br /><searchLink fieldCode="AU" term="%22Bagel+J%22">Bagel J</searchLink>; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Shyng+C%22">Shyng C</searchLink>; Gene Therapy Center, University of North Carolina - Chapel Hill, Chapel Hill, NC 27599, USA.<br /><searchLink fieldCode="AU" term="%22Odonnell+P%22">Odonnell P</searchLink>; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Miyadera+K%22">Miyadera K</searchLink>; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Srnak+J%22">Srnak J</searchLink>; M6P Therapeutics, St. Louis, MO 63108, USA.<br /><searchLink fieldCode="AU" term="%22Swain+G%22">Swain G</searchLink>; Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Pesayco+JP%22">Pesayco JP</searchLink>; Department of Pathology, School of Medicine, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Shelton+GD%22">Shelton GD</searchLink>; Department of Pathology, School of Medicine, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Assenmacher+CA%22">Assenmacher CA</searchLink>; Comparative Pathology Core, Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Dickson+P%22">Dickson P</searchLink>; Department of Pediatrics, Washington University, St. Louis, MO 63110, USA.<br /><searchLink fieldCode="AU" term="%22Stern+JA%22">Stern JA</searchLink>; Department of Clinical Sciences, College of Veterinary Medicine, North Carolina State University, Raleigh, NC 27607, USA.<br /><searchLink fieldCode="AU" term="%22Flanagan-Steet+H%22">Flanagan-Steet H</searchLink>; JC Self Research Institute, Greenwood Genetic Center, Greenwood, SC 29646, USA.<br /><searchLink fieldCode="AU" term="%22Gray+SJ%22">Gray SJ</searchLink>; Gene Therapy Center, University of North Carolina - Chapel Hill, Chapel Hill, NC 27599, USA.<br /><searchLink fieldCode="AU" term="%22Bradbury+AM%22">Bradbury AM</searchLink>; 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.
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