MafA synthetic modified mRNA

MafA synthetic modified mRNA is an experimental mRNA-based therapy designed to reprogram human pancreatic duct-derived cells into functional, insulin-secreting β-like cells for the potential treatment of type 1 diabetes. It encodes the V-Maf musculoaponeurotic fibrosarcoma oncogene homolog A (MAFA) transcription factor, which is a critical regulator of insulin gene expression and β-cell maturation. The mRNA is chemically modified with 5-methylcytidine and pseudouridine to minimize innate immune activation and enhance translation efficiency. In preclinical studies, cells treated with this modified mRNA demonstrated glucose-dependent insulin secretion and successfully reduced blood glucose levels upon transplantation into diabetic animal models. The therapy works by activating a network of genes essential for β-cell function, including insulin (INS), pyruvate carboxylase (PC), and the prolactin receptor (PRLR).

Other names

MAFA mRNAmodified MAFA mRNA