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Showing 1–3 of 3 results
Advanced filters: Author: Kerry E. Benenato Clear advanced filters

  • Propionic acidemia is a serious pediatric inherited disorder with no effective treatments. Here the authors demonstrate that delivering dual mRNAs as an enzyme replacement approach can be used as an effective therapy in a mouse model of propionic acidemia, with potential applicability to chronically administer multiple mRNAs in other genetic disorders.

    • Lei Jiang
    • Ji-Sun Park
    • Lin T. Guey
    ResearchOpen Access
    Nature Communications
    Volume: 11, P: 1-10

  • Drug-target residence time is viewed as a predictor of the clinical efficacy of small-molecule drugs. A pharmacodynamic model, taking into account the target binding kinetics of antibacterial compounds, leads to accurate predictions of cellular and in vivo efficacies of the inhibitors.

    • Grant K Walkup
    • Zhiping You
    • Stewart L Fisher
    Research
    Nature Chemical Biology
    Volume: 11, P: 416-423