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Advanced filters: Author: Kara Hoar 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