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

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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
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  Data: A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies.
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  Data: <searchLink fieldCode="AU" term="%22Dame+HB%22">Dame HB</searchLink>; Genetics, Molecular and Cell Biology Program, Tufts University School of Medicine, Boston, MA, United States.; Tufts University, Department of Biomedical Engineering, Medford, MA, USA.<br /><searchLink fieldCode="AU" term="%22Kopalle+H%22">Kopalle H</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Miñana+B%22">Miñana B</searchLink>; Centre for Genomic Regulation (CRG), The Barcelona Institute of Science and Technology, 08003 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Klaft+Z%22">Klaft Z</searchLink>; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States.<br /><searchLink fieldCode="AU" term="%22Fahey+JB%22">Fahey JB</searchLink>; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States.; Neuroscience Program, Tufts University School of Medicine, Boston, MA, United States.<br /><searchLink fieldCode="AU" term="%22McWatters+DC%22">McWatters DC</searchLink>; Department of Biology, MIT, Cambridge, MA United States.<br /><searchLink fieldCode="AU" term="%22Rao+V%22">Rao V</searchLink>; Sanford Stem Cell Institute and Stem Cell Program, University of California San Diego, La Jolla, CA 92037, United States.<br /><searchLink fieldCode="AU" term="%22Suresh+R%22">Suresh R</searchLink>; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, United States.<br /><searchLink fieldCode="AU" term="%22Aigner+S%22">Aigner S</searchLink>; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, United States.<br /><searchLink fieldCode="AU" term="%22Hammer+MF%22">Hammer MF</searchLink>; BIO5 Institute, University of Arizona, Tucson, Arizona, USA.; Neurology Department, University of Arizona, Tucson, Arizona, USA.<br /><searchLink fieldCode="AU" term="%22Yuskaitis+CJ%22">Yuskaitis CJ</searchLink>; Department of Neurology, FM Kirby Neurobiology Center, Boston Children's Hospital, Harvard Medical School, Boston, USA.<br /><searchLink fieldCode="AU" term="%22Wong+M%22">Wong M</searchLink>; Genetics, Molecular and Cell Biology Program, Tufts University School of Medicine, Boston, MA, United States.<br /><searchLink fieldCode="AU" term="%22Teaney+N%22">Teaney N</searchLink>; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States.; Neuroscience Program, Tufts University School of Medicine, Boston, MA, United States.<br /><searchLink fieldCode="AU" term="%22Zhang+C%22">Zhang C</searchLink>; Genetics, Molecular and Cell Biology Program, Tufts University School of Medicine, Boston, MA, United States.; Tufts University, Department of Biomedical Engineering, Medford, MA, USA.<br /><searchLink fieldCode="AU" term="%22Thompson+M%22">Thompson M</searchLink>; Centre for Genomic Regulation (CRG), The Barcelona Institute of Science and Technology, 08003 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Lehner+B%22">Lehner B</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Bennett+CF%22">Bennett CF</searchLink>; Ionis Pharmaceuticals, Carlsbad, California.<br /><searchLink fieldCode="AU" term="%22Valcárcel+J%22">Valcárcel J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Yeo+GW%22">Yeo GW</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Burge+CB%22">Burge CB</searchLink>; Department of Biology, MIT, Cambridge, MA United States.<br /><searchLink fieldCode="AU" term="%22Dulla+CG%22">Dulla CG</searchLink>; Department of Neuroscience, Tufts University School of Medicine, Boston, MA, United States.<br /><searchLink fieldCode="AU" term="%22Oudin+MJ%22">Oudin MJ</searchLink>; Tufts University, Department of Biomedical Engineering, Medford, MA, USA.
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