Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia
oleh: Lei Jiang, Ji-Sun Park, Ling Yin, Rodrigo Laureano, Eric Jacquinet, Jinsong Yang, Shi Liang, Andrea Frassetto, Jenny Zhuo, Xinhua Yan, Xuling Zhu, Steven Fortucci, Kara Hoar, Cosmin Mihai, Christopher Tunkey, Vlad Presnyak, Kerry E. Benenato, Christine M. Lukacs, Paolo G. V. Martini, Lin T. Guey
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2020-10-01 |
Deskripsi
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.