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FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.

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Title: FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.
Authors: Lacher RK; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Henson K; Forge Biologics, Grove City, OH, USA., Wathen LN; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Jones C; Forge Biologics, Grove City, OH, USA., Arnold T; Forge Biologics, Grove City, OH, USA., Rice MR; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Carles HM; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., White AR; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Ramsuchit E; Forge Biologics, Grove City, OH, USA., Murrey D; Forge Biologics, Grove City, OH, USA., Raig R; Forge Biologics, Grove City, OH, USA., Fisher A; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Bucher K; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Westerkamp GC; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Fritz AL; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Gollaway BM; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Piloto S; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Dismuke D; Forge Biologics, Grove City, OH, USA., Robinson JE; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Williams MT; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA., Vorhees CV; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA., De Silva EK; Forge Biologics, Grove City, OH, USA., Tiwari D; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. durgesh.tiwari@cchmc.org.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. durgesh.tiwari@cchmc.org., Erickson CA; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. craig.erickson@cchmc.org.; Department of Psychiatry, University of Cincinnati College of Medicine, Cincinnati, OH, USA. craig.erickson@cchmc.org., Pedapati EV; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. ernest.pedapati@cchmc.org.; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. ernest.pedapati@cchmc.org.; Department of Psychiatry, University of Cincinnati College of Medicine, Cincinnati, OH, USA. ernest.pedapati@cchmc.org., Gross C; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. christina.gross@cchmc.org.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. christina.gross@cchmc.org.
Source: Gene therapy [Gene Ther] 2026 Jul 10. Date of Electronic Publication: 2026 Jul 10.
Publication Type: Journal Article
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
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  Data: FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome.
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  Data: <searchLink fieldCode="AU" term="%22Lacher+RK%22">Lacher RK</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Henson+K%22">Henson K</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Wathen+LN%22">Wathen LN</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Jones+C%22">Jones C</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Arnold+T%22">Arnold T</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Rice+MR%22">Rice MR</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Carles+HM%22">Carles HM</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22White+AR%22">White AR</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Ramsuchit+E%22">Ramsuchit E</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Murrey+D%22">Murrey D</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Raig+R%22">Raig R</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Fisher+A%22">Fisher A</searchLink>; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Bucher+K%22">Bucher K</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Westerkamp+GC%22">Westerkamp GC</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Fritz+AL%22">Fritz AL</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Gollaway+BM%22">Gollaway BM</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Piloto+S%22">Piloto S</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Dismuke+D%22">Dismuke D</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Robinson+JE%22">Robinson JE</searchLink>; Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Williams+MT%22">Williams MT</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Vorhees+CV%22">Vorhees CV</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22De+Silva+EK%22">De Silva EK</searchLink>; Forge Biologics, Grove City, OH, USA.<br /><searchLink fieldCode="AU" term="%22Tiwari+D%22">Tiwari D</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. durgesh.tiwari@cchmc.org.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. durgesh.tiwari@cchmc.org.<br /><searchLink fieldCode="AU" term="%22Erickson+CA%22">Erickson CA</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. craig.erickson@cchmc.org.; Department of Psychiatry, University of Cincinnati College of Medicine, Cincinnati, OH, USA. craig.erickson@cchmc.org.<br /><searchLink fieldCode="AU" term="%22Pedapati+EV%22">Pedapati EV</searchLink>; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. ernest.pedapati@cchmc.org.; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. ernest.pedapati@cchmc.org.; Department of Psychiatry, University of Cincinnati College of Medicine, Cincinnati, OH, USA. ernest.pedapati@cchmc.org.<br /><searchLink fieldCode="AU" term="%22Gross+C%22">Gross C</searchLink>; Division of Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. christina.gross@cchmc.org.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. christina.gross@cchmc.org.
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