Anti-MUC1 CAR mRNA-LNP

MUC1 (mucin short variant S1), also known as polymorphic epithelial mucin (PEM) or epithelial membrane antigen (EMA), is one of several mucins that make up mucus, a slippery substance that can Lubricate and protect the inner wall of the airway, digestive system, reproductive system and other organs and tissues. In addition to its role in mucus, mucin 1 is also involved in cell signaling and kidney development. MUC1, encoded by the human MUC1 gene, is a glycoprotein with extensive O-linked glycosylation in the extracellular domain. Mucins line the apical surface of epithelial cells in the lungs, stomach, intestines, eyes, and several other organs, protecting these organs from infection by pathogens that bind to oligosaccharides in the extracellular domain. MUC1 was also found to regulate the activity of transcription factor complexes, suggesting a role in tumor-induced changes in host immunity. Overexpression of MUC1 has been reported to be commonly associated with colon, breast, ovarian, lung and pancreatic cancers. This product is designed as a tool for the delivery and expression of anti-MUC1 CAR mRNA for research. The product leverages the lipid nanoparticle (LNP) technology platform for simple and efficient delivery of anti-MUC1 CAR mRNA to a variety of mammalian cells in vitro and in vivo. The LNPs used are formulated with SM-102, DSPC, cholesterol and DMG-PEG2000 at an optimal molar concentration for a high rate of encapsulation and efficient mRNA delivery. The anti-MUC1 CAR in this product is approximately 56 kD and consists of anti-MUC1 scFv (single-chain variable fragment) linked to a second-generation CAR (chimeric antigen receptor) containing the CD8 hinge and transmembrane domain and 4-1BB and CD3ζ signaling domains. The full-length amino acid sequence of this anti-MUC1 CAR mRNA-LNP product is available upon request.