RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I.
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| Title: | RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I. |
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| Authors: | Tálas A; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Ioannidi EI; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Weber Y; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Haenggi T; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Kulcsár PI; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Zürcher N; Division of Metabolism and Children's Research Center, University Children's Hospital Zurich, Zurich, Switzerland., Faccin E; Division of Metabolism and Children's Research Center, University Children's Hospital Zurich, Zurich, Switzerland., Moon WJ; Acuitas Therapeutics Inc., Vancouver, BC, Canada., Kissling L; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Villiger EA; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Matsushita M; Department of Biology, Institute of Molecular Health Sciences, ETH Zurich, Zurich, Switzerland., Rothgangl T; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Janjuha S; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Lin PJC; Acuitas Therapeutics Inc., Vancouver, BC, Canada., Poms M; Division of Clinical Chemistry and Biochemistry, University Children's Hospital Zurich, Zurich, Switzerland., Muramatsu H; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Vadovics M; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Reichmuth A; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland., Kopf M; Department of Biology, Institute of Molecular Health Sciences, ETH Zurich, Zurich, Switzerland., Pardi N; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Tam YK; Acuitas Therapeutics Inc., Vancouver, BC, Canada., Häberle J; Division of Metabolism and Children's Research Center, University Children's Hospital 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. eaec7274. 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 |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1946-6242 |
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| DOI: | 10.1126/scitranslmed.aec7274 |
