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

Saved in:
Bibliographic Details
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
Database: MEDLINE Ultimate
Description
ISSN:1476-5462
DOI:10.1038/s41434-026-00630-4