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 |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 40285357 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II. – Name: Author Label: Authors Group: Au 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. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2025 Aug 06; Vol. 33 (8), pp. 3808-3821. <i>Date of Electronic Publication: </i>2025 Apr 24. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=40285357 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1016/j.ymthe.2025.04.030 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 3808 Titles: – TitleFull: Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Pyne NK – PersonEntity: Name: NameFull: Bagel J – PersonEntity: Name: NameFull: Shyng C – PersonEntity: Name: NameFull: Odonnell P – PersonEntity: Name: NameFull: Miyadera K – PersonEntity: Name: NameFull: Srnak J – PersonEntity: Name: NameFull: Swain G – PersonEntity: Name: NameFull: Pesayco JP – PersonEntity: Name: NameFull: Shelton GD – PersonEntity: Name: NameFull: Assenmacher CA – PersonEntity: Name: NameFull: Dickson P – PersonEntity: Name: NameFull: Stern JA – PersonEntity: Name: NameFull: Flanagan-Steet H – PersonEntity: Name: NameFull: Gray SJ – PersonEntity: Name: NameFull: Bradbury AM IsPartOfRelationships: – BibEntity: Dates: – D: 06 M: 08 Text: 2025 Aug 06 Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 1525-0024 Numbering: – Type: volume Value: 33 – Type: issue Value: 8 Titles: – TitleFull: Molecular therapy : the journal of the American Society of Gene Therapy Type: main |
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