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Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.

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Title: Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
Authors: Herstine JA; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA; Center for Clinical and Translational Science, The Ohio State University, Columbus, OH 43210, USA., Chang PK; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA., Chornyy S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA., Stevenson TJ; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA., Sunshine AC; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Neurology, Nationwide Children's Hospital, Columbus, OH 43205, USA., Nokhrina K; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA., Rediger J; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA., Wentz J; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA., Vetter TA; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA., Scholl E; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA., Holaway C; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA., Pyne NK; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA., Bratasz A; Small Animal Imaging Core, The Ohio State University, Columbus, OH 43210, USA., Yeoh S; Preclinical Imaging Core, The University of Utah, Salt Lake City, UT 84112, USA., Flanigan KM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA., Bonkowsky JL; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA; Center for Personalized Medicine, Primary Children's Hospital, Salt Lake City, UT 84113, USA. Electronic address: joshua.bonkowsky@hsc.utah.edu., Bradbury AM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA; Center for Clinical and Translational Science, The Ohio State University, Columbus, OH 43210, USA. Electronic address: allison.bradbury@nationwidechildrens.org.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Jun 05; Vol. 32 (6), pp. 1701-1720. Date of Electronic Publication: 2024 Mar 27.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
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: Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
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  Data: <searchLink fieldCode="AU" term="%22Herstine+JA%22">Herstine JA</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA; Center for Clinical and Translational Science, The Ohio State University, Columbus, OH 43210, USA.<br /><searchLink fieldCode="AU" term="%22Chang+PK%22">Chang PK</searchLink>; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA.<br /><searchLink fieldCode="AU" term="%22Chornyy+S%22">Chornyy S</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA.<br /><searchLink fieldCode="AU" term="%22Stevenson+TJ%22">Stevenson TJ</searchLink>; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA.<br /><searchLink fieldCode="AU" term="%22Sunshine+AC%22">Sunshine AC</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Neurology, Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Nokhrina+K%22">Nokhrina K</searchLink>; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA.<br /><searchLink fieldCode="AU" term="%22Rediger+J%22">Rediger J</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA.<br /><searchLink fieldCode="AU" term="%22Wentz+J%22">Wentz J</searchLink>; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA.<br /><searchLink fieldCode="AU" term="%22Vetter+TA%22">Vetter TA</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.<br /><searchLink fieldCode="AU" term="%22Scholl+E%22">Scholl E</searchLink>; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA.<br /><searchLink fieldCode="AU" term="%22Holaway+C%22">Holaway C</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA.<br /><searchLink fieldCode="AU" term="%22Pyne+NK%22">Pyne NK</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA.<br /><searchLink fieldCode="AU" term="%22Bratasz+A%22">Bratasz A</searchLink>; Small Animal Imaging Core, The Ohio State University, Columbus, OH 43210, USA.<br /><searchLink fieldCode="AU" term="%22Yeoh+S%22">Yeoh S</searchLink>; Preclinical Imaging Core, The University of Utah, Salt Lake City, UT 84112, USA.<br /><searchLink fieldCode="AU" term="%22Flanigan+KM%22">Flanigan KM</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.<br /><searchLink fieldCode="AU" term="%22Bonkowsky+JL%22">Bonkowsky JL</searchLink>; Department of Pediatrics, The University of Utah School of Medicine, Salt Lake City, UT 84113, USA; Center for Personalized Medicine, Primary Children's Hospital, Salt Lake City, UT 84113, USA. Electronic address: joshua.bonkowsky@hsc.utah.edu.<br /><searchLink fieldCode="AU" term="%22Bradbury+AM%22">Bradbury AM</searchLink>; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA; Center for Clinical and Translational Science, The Ohio State University, Columbus, OH 43210, USA. Electronic address: allison.bradbury@nationwidechildrens.org.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2024 Jun 05; Vol. 32 (6), pp. 1701-1720. <i>Date of Electronic Publication: </i>2024 Mar 27.
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  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
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        Value: 10.1016/j.ymthe.2024.03.034
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              Text: 2024 Jun 05
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