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Current clinical applications of AAV-mediated gene therapy.

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Title: Current clinical applications of AAV-mediated gene therapy.
Authors: Byrne BJ; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA. Electronic address: barry.byrne@ufl.edu., Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA., Matesanz SE; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Philadelphia, PA, USA., Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA., Waldrop MA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA., D'Ambrosio ES; Department of Neurology, University of Massachusetts Chan School of Medicine, Worcester, MA, USA., Johnson NE; Department of Neurology and Center for Inherited Muscle Research, Virginia Commonwealth University, Richmond, VA, USA., Smith BK; Department of Physical Therapy, College of Public Health and Health Professions, University of Florida Gainesville, FL, USA., Bönnemann C; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA., Carrig S; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Rossano JW; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA; Division of Cardiology, Children's Hospital of Philadelphia, Philadelphia PA, USA., Greenberg B; Cardiology Department, University of California, California, San Diego, La Jolla, CA, USA., Lalaguna L; Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), Madrid, Spain., Lara-Pezzi E; Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), Madrid, Spain; Centro de Investigación Biomédica en Red Cardiovascular (CIBERCV), Madrid, Spain., Subramony S; Department of Neurology, University of Florida College of Medicinem, Gainesville, FL, USA., Corti M; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA., Mercado-Rodriguez C; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA., Leon-Astudillo C; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA., Ahrens-Nicklas R; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA., Bharucha-Goebel D; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA; Division of Neurology, Children's National Hospital, Washington, DC, USA., Gao G; Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan School of Medicine, Worcester, MA, USA., Gessler DJ; Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan School of Medicine, Worcester, MA, USA., Hwu WL; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan; Center for Precision Medicine, China Medical University Hospital, Taichung, Taiwan., Chien YH; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan., Lee NC; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan., Boye SL; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA., Boye SE; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA., George LA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA. Electronic address: georgel@chop.edu.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Jun 04; Vol. 33 (6), pp. 2479-2516. Date of Electronic Publication: 2025 May 05.
Publication Type: Journal Article; Review
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: Current clinical applications of AAV-mediated gene therapy.
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  Data: <searchLink fieldCode="AU" term="%22Byrne+BJ%22">Byrne BJ</searchLink>; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA. Electronic address: barry.byrne@ufl.edu.<br /><searchLink fieldCode="AU" term="%22Flanigan+KM%22">Flanigan KM</searchLink>; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.<br /><searchLink fieldCode="AU" term="%22Matesanz+SE%22">Matesanz SE</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Finkel+RS%22">Finkel RS</searchLink>; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.<br /><searchLink fieldCode="AU" term="%22Waldrop+MA%22">Waldrop MA</searchLink>; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.<br /><searchLink fieldCode="AU" term="%22D'Ambrosio+ES%22">D'Ambrosio ES</searchLink>; Department of Neurology, University of Massachusetts Chan School of Medicine, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Johnson+NE%22">Johnson NE</searchLink>; Department of Neurology and Center for Inherited Muscle Research, Virginia Commonwealth University, Richmond, VA, USA.<br /><searchLink fieldCode="AU" term="%22Smith+BK%22">Smith BK</searchLink>; Department of Physical Therapy, College of Public Health and Health Professions, University of Florida Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Bönnemann+C%22">Bönnemann C</searchLink>; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Carrig+S%22">Carrig S</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Rossano+JW%22">Rossano JW</searchLink>; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA; Division of Cardiology, Children's Hospital of Philadelphia, Philadelphia PA, USA.<br /><searchLink fieldCode="AU" term="%22Greenberg+B%22">Greenberg B</searchLink>; Cardiology Department, University of California, California, San Diego, La Jolla, CA, USA.<br /><searchLink fieldCode="AU" term="%22Lalaguna+L%22">Lalaguna L</searchLink>; Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Lara-Pezzi+E%22">Lara-Pezzi E</searchLink>; Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC), Madrid, Spain; Centro de Investigación Biomédica en Red Cardiovascular (CIBERCV), Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Subramony+S%22">Subramony S</searchLink>; Department of Neurology, University of Florida College of Medicinem, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Corti+M%22">Corti M</searchLink>; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Mercado-Rodriguez+C%22">Mercado-Rodriguez C</searchLink>; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Leon-Astudillo+C%22">Leon-Astudillo C</searchLink>; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Ahrens-Nicklas+R%22">Ahrens-Nicklas R</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Bharucha-Goebel+D%22">Bharucha-Goebel D</searchLink>; Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA; Division of Neurology, Children's National Hospital, Washington, DC, USA.<br /><searchLink fieldCode="AU" term="%22Gao+G%22">Gao G</searchLink>; Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan School of Medicine, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Gessler+DJ%22">Gessler DJ</searchLink>; Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan School of Medicine, Worcester, MA, USA.<br /><searchLink fieldCode="AU" term="%22Hwu+WL%22">Hwu WL</searchLink>; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan; Center for Precision Medicine, China Medical University Hospital, Taichung, Taiwan.<br /><searchLink fieldCode="AU" term="%22Chien+YH%22">Chien YH</searchLink>; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Lee+NC%22">Lee NC</searchLink>; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, and College of Medicine, National Taiwan University, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Boye+SL%22">Boye SL</searchLink>; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Boye+SE%22">Boye SE</searchLink>; Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22George+LA%22">George LA</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA, USA. Electronic address: georgel@chop.edu.
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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] 2025 Jun 04; Vol. 33 (6), pp. 2479-2516. <i>Date of Electronic Publication: </i>2025 May 05.
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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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