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

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.... show more
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Citations
67
Influential Citations
1
Reference Count
70
Citation by Year

Note: The citation metrics presented here have been sourced from Semantic Scholar and OpenAlex.

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