Nature Communications (May 2021)
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease
- Jingsong Cao,
- Minjung Choi,
- Eleonora Guadagnin,
- Maud Soty,
- Marine Silva,
- Vincent Verzieux,
- Edward Weisser,
- Arianna Markel,
- Jenny Zhuo,
- Shi Liang,
- Ling Yin,
- Andrea Frassetto,
- Anne-Renee Graham,
- Kristine Burke,
- Tatiana Ketova,
- Cosmin Mihai,
- Zach Zalinger,
- Becca Levy,
- Gilles Besin,
- Meredith Wolfrom,
- Barbara Tran,
- Christopher Tunkey,
- Erik Owen,
- Joe Sarkis,
- Athanasios Dousis,
- Vladimir Presnyak,
- Christopher Pepin,
- Wei Zheng,
- Lei Ci,
- Marjie Hard,
- Edward Miracco,
- Lisa Rice,
- Vi Nguyen,
- Mike Zimmer,
- Uma Rajarajacholan,
- Patrick F. Finn,
- Gilles Mithieux,
- Fabienne Rajas,
- Paolo G. V. Martini,
- Paloma H. Giangrande
Affiliations
- Jingsong Cao
- Rare Diseases, Moderna, Inc
- Minjung Choi
- Rare Diseases, Moderna, Inc
- Eleonora Guadagnin
- Rare Diseases, Moderna, Inc
- Maud Soty
- INSERM UMR1213, Université Claude Bernard Lyon 1
- Marine Silva
- INSERM UMR1213, Université Claude Bernard Lyon 1
- Vincent Verzieux
- INSERM UMR1213, Université Claude Bernard Lyon 1
- Edward Weisser
- Rare Diseases, Moderna, Inc
- Arianna Markel
- Rare Diseases, Moderna, Inc
- Jenny Zhuo
- Rare Diseases, Moderna, Inc
- Shi Liang
- Rare Diseases, Moderna, Inc
- Ling Yin
- Rare Diseases, Moderna, Inc
- Andrea Frassetto
- Rare Diseases, Moderna, Inc
- Anne-Renee Graham
- Platform, Moderna, Inc
- Kristine Burke
- Platform, Moderna, Inc
- Tatiana Ketova
- Platform, Moderna, Inc
- Cosmin Mihai
- Platform, Moderna, Inc
- Zach Zalinger
- Platform, Moderna, Inc
- Becca Levy
- Platform, Moderna, Inc
- Gilles Besin
- Platform, Moderna, Inc
- Meredith Wolfrom
- Platform, Moderna, Inc
- Barbara Tran
- Platform, Moderna, Inc
- Christopher Tunkey
- Platform, Moderna, Inc
- Erik Owen
- Platform, Moderna, Inc
- Joe Sarkis
- Platform, Moderna, Inc
- Athanasios Dousis
- Platform, Moderna, Inc
- Vladimir Presnyak
- Platform, Moderna, Inc
- Christopher Pepin
- Platform, Moderna, Inc
- Wei Zheng
- Platform, Moderna, Inc
- Lei Ci
- Platform, Moderna, Inc
- Marjie Hard
- Platform, Moderna, Inc
- Edward Miracco
- Platform, Moderna, Inc
- Lisa Rice
- Rare Diseases, Moderna, Inc
- Vi Nguyen
- Rare Diseases, Moderna, Inc
- Mike Zimmer
- Rare Diseases, Moderna, Inc
- Uma Rajarajacholan
- Rare Diseases, Moderna, Inc
- Patrick F. Finn
- Rare Diseases, Moderna, Inc
- Gilles Mithieux
- INSERM UMR1213, Université Claude Bernard Lyon 1
- Fabienne Rajas
- INSERM UMR1213, Université Claude Bernard Lyon 1
- Paolo G. V. Martini
- Rare Diseases, Moderna, Inc
- Paloma H. Giangrande
- Rare Diseases, Moderna, Inc
- DOI
- https://doi.org/10.1038/s41467-021-23318-2
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 14
Abstract
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.
