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
Description
ISSN:1525-0024
DOI:10.1016/j.ymthe.2024.03.034