RNA-LNP–mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I
. These findings highlight prime editing as a precise and potentially curative treatment strategy for individuals with CTLN1 and other genetic liver diseases.
Authors
- Gerald Schwank (ORCID: https://orcid.org/0000-0003-0767-2953)
- Paulo J.C. Lin
- Sharan Janjuha (ORCID: https://orcid.org/0000-0002-5910-2912)
- Péter István Kulcsár (ORCID: https://orcid.org/0000-0002-7783-5108)
- Tanja Rothgangl (ORCID: https://orcid.org/0000-0001-7799-2413)
- Mai Matsushita (ORCID: https://orcid.org/0000-0001-5693-461X)
- Tatjana Haenggi
- Norbert Pardi (ORCID: https://orcid.org/0000-0003-1008-6242)
- Martin Poms (ORCID: https://orcid.org/0000-0002-4426-314X)
- Yanik Weber (ORCID: https://orcid.org/0000-0002-0914-8541)
- Hiromi Muramatsu (ORCID: https://orcid.org/0000-0003-1544-1493)
- András Tálas (ORCID: https://orcid.org/0000-0003-0851-8333)
- Máté Vadovics (ORCID: https://orcid.org/0000-0001-9028-987X)
- Erica Faccin (ORCID: https://orcid.org/0000-0002-8817-4923)
- Lucas Kissling (ORCID: https://orcid.org/0000-0002-4989-0695)
- Nadia Zürcher
- Elina Andrea Villiger
- Ying K. Tam
- Manfred Kopf
- Eleonora I. Ioannidi
- Johannes Häberle (ORCID: https://orcid.org/0000-0003-0635-091X)
- Andreas Reichmuth (ORCID: https://orcid.org/0000-0002-4365-2119)
- Woohyun J. Moon (ORCID: https://orcid.org/0009-0001-1395-823X)
Institutions
- University of Zurich (CH)
- ETH Zurich (CH)
- University Children's Hospital Zurich (CH)
- Acuitas Therapeutics (Canada) (CA)
- University of Pennsylvania (US)
Publication Details
- Journal
- Science Translational Medicine
- Published
- 2026-05-13
- DOI
- https://doi.org/10.1126/scitranslmed.aec7274
- Primary Topic
- Metabolism and Genetic Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Schweizerischer Nationalfonds zur Förderung der Wissenschaftlichen Forschung
- Citrin Foundation