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....
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Nature Portfolio
2021
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oai:doaj.org-article:977236f43e9f4fb4a8b6a0322537f1cb2021-12-02T14:49:22ZmRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease10.1038/s41467-021-23318-22041-1723https://doaj.org/article/977236f43e9f4fb4a8b6a0322537f1cb2021-05-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-23318-2https://doaj.org/toc/2041-1723Glycogen 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.Jingsong CaoMinjung ChoiEleonora GuadagninMaud SotyMarine SilvaVincent VerzieuxEdward WeisserArianna MarkelJenny ZhuoShi LiangLing YinAndrea FrassettoAnne-Renee GrahamKristine BurkeTatiana KetovaCosmin MihaiZach ZalingerBecca LevyGilles BesinMeredith WolfromBarbara TranChristopher TunkeyErik OwenJoe SarkisAthanasios DousisVladimir PresnyakChristopher PepinWei ZhengLei CiMarjie HardEdward MiraccoLisa RiceVi NguyenMike ZimmerUma RajarajacholanPatrick F. FinnGilles MithieuxFabienne RajasPaolo G. V. MartiniPaloma H. GiangrandeNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-14 (2021) |
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DOAJ |
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EN |
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Science Q |
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Science Q 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 mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| description |
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. |
| format |
article |
| author |
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 |
| author_facet |
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 |
| author_sort |
Jingsong Cao |
| title |
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| title_short |
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| title_full |
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| title_fullStr |
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| title_full_unstemmed |
mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| title_sort |
mrna therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease |
| publisher |
Nature Portfolio |
| publishDate |
2021 |
| url |
https://doaj.org/article/977236f43e9f4fb4a8b6a0322537f1cb |
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