Medicine and Health

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

J. Cao, M. Choi, et al.

This groundbreaking study conducted by a team of experts reveals a promising new therapy for Glycogen Storage Disease 1a (GSD1a), a rare metabolic disorder caused by glucose 6-phosphatase deficiency. By utilizing engineered mRNAs in lipid nanoparticles, they demonstrated significant efficacy and safety in a murine model, opening doors to potential treatment where none currently exists.

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Abstract

Glycogen Storage Disease 1a (GSD1a) is a rare, inherited metabolic disorder caused by deficiency of glucose 6-phosphatase (G6Pase-α). GSD1a patients exhibit life-threatening hypoglycemia and long-term liver complications including hepatocellular adenomas (HCAs) and carcinomas (HCCs). There is no effective treatment. This study encapsulated engineered mRNAs encoding human G6Pase-α in lipid nanoparticles and demonstrated efficacy and safety in a murine model, presenting a potential new therapy.

Publisher

Nature Communications

Published On

May 25, 2021

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, Lisa Rice, Vi Nguyen, Mike Zimmer, Uma Rajarajacholan, Patrick F. Finn, Gilles Mithieux, Fabienne Rajas, Paolo G. V. Martini, Paloma H. Giangrande

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