FIGURE 1.

Schematic illustration of mRNA therapeutics. (A) The central dogma. mRNA therapeutics work through the expression of functional proteins or peptides in cytoplasm. (B) For disease prophylaxis or immunotherapy, mRNA encoding an antigen is internalized by somatic cells (e.g., muscle cells) or antigen‐presenting cells (APCs) after intramuscular injection. Then, antigens expressed in the cytoplasm are degraded by proteasomes, and then effector cells (e.g., T cells and B cells) are activated to detect and eradicate pathogens directly. The therapeutic antibodies can also be produced by mRNA for passive immunity. (C) mRNA has been used for protein replacement therapy, encoding transmembrane, intracellular, and secreted proteins. (D) mRNA can be applied to encode the Cas9 protein for gene editing in vivo. (E) mRNA encoding reprogramming factors can reprogram cells into induced pluripotent stem cells in vitro, which can differentiate into desired functional cells for tissue regeneration.