Glycogen synthase 2 (GYS2) mRNA (GYS2 mRNA)

Glycogen synthase 2 (GYS2) mRNA is the transcript encoding the liver-specific isoform of glycogen synthase, the rate-limiting enzyme responsible for converting glucose into glycogen (UniProt P54840) [1]. In the liver, GYS2 is critical for maintaining glucose homeostasis by storing excess glucose after meals and providing a reservoir for glucose release during fasting (NCBI Gene ID: 2998) [2]. Mutations in the GYS2 gene lead to Glycogen Storage Disease type 0a (GSD 0a), a condition characterized by the inability to store liver glycogen, resulting in severe fasting hypoglycemia and postprandial hyperglycemia (NIH GARD) [3]. As a therapeutic target, GYS2 mRNA is utilized in mRNA replacement therapies where synthetic mRNA encapsulated in lipid nanoparticles is delivered to the liver to restore enzyme function [4]. This approach aims to normalize glucose metabolism and prevent the metabolic crises associated with GSD 0a [5]. Current research focuses on optimizing delivery systems to ensure efficient hepatocyte uptake and sustained protein expression while minimizing potential immune responses to the mRNA-LNP complex [4].

Other names

Liver glycogen synthase mRNAGYS2 transcriptGlycogen synthase 2 (liver)

mRNA replacement therapy: Delivery of exogenous mRNA to hepatocytes via lipid nanoparticles (LNPs) to enable the translation of functional Glycogen Synthase 2 protein, thereby restoring the ability to synthesize liver glycogen [4].