mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease

mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease

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....

Full description

Saved in:
Bibliographic Details
Main Authors: 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
Format: article
Language:EN
Published: Nature Portfolio 2021
Subjects:
Science
Q
Online Access:https://doaj.org/article/977236f43e9f4fb4a8b6a0322537f1cb
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items

Similar Items