GeneticsbioRxivPreprint
mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria
Sonam Gurung, Oskar Vilhelmsson Timmermand, Dany P.B. Perocheau…·Julien Baruteau
This study shows that mRNA therapy restores ureagenesis and glutathione metabolism in ASL-deficient mice, correcting liver disease and neurocognitive impairments.
10 citations