A polynucleotide encoding glucose-6-phosphatase for treating glycogen disease

摘要

This disclosure relates to an mRNA therapeutic agent for treating related diseases such as glycogen disease type 1a, (GSD-Ia), and hypoglycemia.The mRNA used in the present invention is administered in vivo, encoding a human glucose-6-phosphatase (G6P or g6pc), and its functional fragments and variants.The mRNA of the present invention is preferably encapsulated in lipid nanoparticles (LNP) to provide efficient delivery to the target cells and / or tissue upon administration.The mRNA therapeutic agent of the present invention increases and / or restores the deficiency level of g6pc expression and / or activity in the subject.The mRNA therapeutics of the present invention further increase glucose production and reduce abnormal accumulation of glycogen and / or glucose-6-phosphate related to gss-ia.Diagram