Gene Replacement mRNA

Gene replacement is a popular application of mRNA. Many proteins cannot be replaced by exogenous protein delivery, for example, if they are intracellular, membrane-bound, or post-translationally modified or processed. In vitro transcribed mRNAs encoding physiologically important proteins have considerable potential for therapeutic applications. Expressing proteins by delivering encoded mRNA has many advantages over methods using plasmid DNA or viral vectors. During mRNA transfection, the coding sequence for the desired protein is the only material delivered to the cell, thus avoiding all the side effects associated with plasmid backbones, viral genes, and viral proteins.However, mRNAs are inherently unstable, translationally inefficient, and immunogenic, making them traditionally unsuitable for therapy. Techniques for modifying mRNAs by incorporating non-canonical nucleosides such as pseudouridines increase the translational efficiency and overall stability of mRNAs and reduce their immunogenicity in vivo. These favorable new properties provide an opportunity to develop deliverable pseudouridine-modified mRNAs as vectors for the safe and efficient expression of clinically beneficial proteins.

Human erythropoietin (EPO) protein is a secreted protein that can be easily measured in serum by ELISA. When sequence-engineered mRNA encoding firefly luciferase and EPO is transfected into cells, mRNA produced with regular nucleotides produces more protein than modified nucleotides, so EPO mRNA can be used to mimic your secreted protein.

Creative Biogene produces native Cap 1 structures with high capping efficiency that are polyadenylated, substituted with modified uridines, and optimized for mammalian systems that mimic fully processed mature mRNA. Please browse our products to find what you're looking for.