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Showing 1–2 of 2 results
Advanced filters: Author: Iolanda Boffa Clear advanced filters

  • Ferriero et al. show that liver fibrosis reduces the efficiency of AAV-based gene therapy by limiting liver uptake and altering vector distribution to other organs. The impact of fibrosis varies depending on the AAV variant, highlighting the need for tailored approaches in fibrotic diseases.

    • Rosa Ferriero
    • Gemma Bruno
    • Pasquale Piccolo
    ResearchOpen Access
    Nature Communications
    Volume: 16, P: 1-13

  • Hyperammonemia occurs in liver diseases affecting ureagenesis, and is life-threatening. Here, the authors show that liver UDP-GlcNAc is increased during hyperammonemia, leading to O-GlcNAcylation of the rate-limiting ureagenesis enzyme CPS1, that enhanced ureagenesis and ammonia detoxification. They also showed that pharmacological increase of protein O-GlcNAcylation reduces hyperammonemia in mouse models of liver disease.

    • Leandro R. Soria
    • Georgios Makris
    • Nicola Brunetti-Pierri
    ResearchOpen Access
    Nature Communications
    Volume: 13, P: 1-14