A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies.

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Bibliographic Details
Title: A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies.
Authors: Dame HB; Genetics, Molecular and Cell Biology Program, Tufts University School of Medicine, Boston, MA, United States.; Tufts University, Department of Biomedical Engineering, Medford, MA, USA., Kopalle H; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, United States.; Neurobiology Department, School of Biological Sciences, University of California San Diego, La Jolla, CA, United States.; Sanford Stem Cell Institute and Stem Cell Program, University of California San Diego, La Jolla, CA 92037, United States., Miñana B; Centre for Genomic Regulation (CRG), The Barcelona Institute of Science and Technology, 08003 Barcelona, Spain., Klaft Z; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States., Fahey JB; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States.; Neuroscience Program, Tufts University School of Medicine, Boston, MA, United States., McWatters DC; Department of Biology, MIT, Cambridge, MA United States., Rao V; Sanford Stem Cell Institute and Stem Cell Program, University of California San Diego, La Jolla, CA 92037, United States., Suresh R; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, United States., Aigner S; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, United States., Hammer MF; BIO5 Institute, University of Arizona, Tucson, Arizona, USA.; Neurology Department, University of Arizona, Tucson, Arizona, USA., Yuskaitis CJ; Department of Neurology, FM Kirby Neurobiology Center, Boston Children's Hospital, Harvard Medical School, Boston, USA., Wong M; Genetics, Molecular and Cell Biology Program, Tufts University School of Medicine, Boston, MA, United States., Teaney N; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States.; Neuroscience Program, Tufts University School of Medicine, Boston, MA, United States., Zhang C; Genetics, Molecular and Cell Biology Program, Tufts University School of Medicine, Boston, MA, United States.; Tufts University, Department of Biomedical Engineering, Medford, MA, USA., Thompson M; Centre for Genomic Regulation (CRG), The Barcelona Institute of Science and Technology, 08003 Barcelona, Spain., Lehner B; Centre for Genomic Regulation (CRG), The Barcelona Institute of Science and Technology, 08003 Barcelona, Spain.; Wellcome Sanger Institute, Wellcome Genome Campus, Hinxton, UK.; Institució Catalana de Recerca i Estudis Avançats (ICREA), Barcelona, Spain., Bennett CF; Ionis Pharmaceuticals, Carlsbad, California., Valcárcel J; Centre for Genomic Regulation (CRG), The Barcelona Institute of Science and Technology, 08003 Barcelona, Spain.; Institució Catalana de Recerca i Estudis Avançats (ICREA), Barcelona, Spain.; Universitat Pompeu Fabra (UPF), Barcelona, Spain., Yeo GW; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, United States.; Neurobiology Department, School of Biological Sciences, University of California San Diego, La Jolla, CA, United States.; Sanford Stem Cell Institute and Stem Cell Program, University of California San Diego, La Jolla, CA 92037, United States.; Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, United States.; Center for RNA Technologies & Therapeutics, University of California San Diego, La Jolla, CA 92093, USA.; Sanford Laboratories for Innovative Medicines, La Jolla, CA, United States., Burge CB; Department of Biology, MIT, Cambridge, MA United States., Dulla CG; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States., Oudin MJ; Tufts University, Department of Biomedical Engineering, Medford, MA, USA.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 Oct 22. Date of Electronic Publication: 2025 Oct 22.
Publication Type: Journal Article; Preprint
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2692-8205
DOI:10.1101/2025.10.22.683934