RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I.

doi:10.1126/scitranslmed.aec7274

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Article

RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I.

Science translational medicine - 13 May 2026

Tálas András, Ioannidi Eleonora I, Weber Yanik, Haenggi Tatjana, Kulcsár Péter István, Zürcher Nadia, Faccin Erica, Moon Woohyun J, Kissling Lucas, Villiger Elina Andrea, Matsushita Mai, Rothgangl Tanja, Janjuha Sharan, Lin Paulo J C, Poms Martin, Muramatsu Hiromi, Vadovics Máté, Reichmuth Andreas, Kopf Manfred, Pardi Norbert, Tam Ying K, Häberle Johannes, Schwank Gerald

Abstract excerpt

Citrullinemia type I (CTLN1) is a severe urea cycle disorder caused by pathogenic variants in the ASS1 (argininosuccinate synthetase 1) gene, for which liver transplantation remains the only curative option. Here, we used prime editing to correct the Ass1fold mouse model of CTLN1. Adeno-associated virus (AAV)-mediated delivery of the PE7 prime editor with an optimized prime editing guide RNA (pegRNA) achieved 71...

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PMID: 42127219
DOI: 10.1126/scitranslmed.aec7274

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ASS1

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