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Prime editing of a pathogenic Scn1a allele ameliorates seizure phenotypes in a GEFS+ mouse model.

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Title: Prime editing of a pathogenic Scn1a allele ameliorates seizure phenotypes in a GEFS+ mouse model.
Authors: Kissling L; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Pietrafesa F; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Ranucci M; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Böck D; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Mathis N; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Kulcsár PI; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Ioannidi E; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Schmidheini L; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Tálas A; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Villiger E; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Wildner H; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Zhao X; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Kompotis K; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Zeilhofer HU; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland.; Institute of Pharmaceutical Sciences, Swiss Federal Institute of Technology (ETH) Zurich, Zurich, Switzerland., Schwank G; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland.
Source: Science translational medicine [Sci Transl Med] 2026 May 13; Vol. 18 (849), pp. eadz2557. Date of Electronic Publication: 2026 May 13.
Publication Type: Journal Article
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
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  Data: Prime editing of a pathogenic Scn1a allele ameliorates seizure phenotypes in a GEFS<superscript>+</superscript> mouse model.
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