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RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I
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 Ass1(fold) mouse model of CTLN1. Adeno-associated
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