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

Glycogen Storage Disease 1a (GSD1a) is a rare, inherited metabolic disorder caused by deficiency of glucose 6-phosphatase (G6Pase-α). G6Pase-α is critical for maintaining interprandial euglycemia. GSD1a patients exhibit life-threatening hypoglycemia and long-term liver complications including hepato...

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Detalles Bibliográficos
Autores principales: Cao, Jingsong, Choi, Minjung, Guadagnin, Eleonora, Soty, Maud, Silva, Marine, Verzieux, Vincent, Weisser, Edward, Markel, Arianna, Zhuo, Jenny, Liang, Shi, Yin, Ling, Frassetto, Andrea, Graham, Anne-Renee, Burke, Kristine, Ketova, Tatiana, Mihai, Cosmin, Zalinger, Zach, Levy, Becca, Besin, Gilles, Wolfrom, Meredith, Tran, Barbara, Tunkey, Christopher, Owen, Erik, Sarkis, Joe, Dousis, Athanasios, Presnyak, Vladimir, Pepin, Christopher, Zheng, Wei, Ci, Lei, Hard, Marjie, Miracco, Edward, Rice, Lisa, Nguyen, Vi, Zimmer, Mike, Rajarajacholan, Uma, Finn, Patrick F., Mithieux, Gilles, Rajas, Fabienne, Martini, Paolo G. V., Giangrande, Paloma H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
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Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8149455/
https://www.ncbi.nlm.nih.gov/pubmed/34035281
http://dx.doi.org/10.1038/s41467-021-23318-2

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