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Showing 1–3 of 3 results
Advanced filters: Author: Christopher Tunkey Clear advanced filters

  • Glycogen Storage Disease 1a (Gsd1a) is an inherited disorder caused by glucose 6-phosphatase (G6Pase-α) deficiency and characterized by hypoglycaemia and high risk of liver cancer. Here the authors develop a mRNA-based G6Pase-α delivery therapy that is efficacious and safe in a mouse model of GSD1a.

    • Jingsong Cao
    • Minjung Choi
    • Paloma H. Giangrande
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-14

  • 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

  • IL-2 has been used to expand regulatory T (Treg) cells for treating inflammatory disorders. Here the authors test an engineered IL-2 mutein, delivered subcutaneously as mRNA, to show its increased specificity for activating and expanding Treg cells in both rodents and non-human primates, and to demonstrate its ability to suppress autoimmunity in mouse models.

    • Seymour de Picciotto
    • Nicholas DeVita
    • Eric Huang
    ResearchOpen Access
    Nature Communications
    Volume: 13, P: 1-14