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Nucleic acids encoding protein therapeutics refer to DNA or RNA molecules that serve as templates for the cellular production of therapeutic proteins after delivery into patient cells. Unlike conventional biologic drugs, where the protein itself is administered, these nucleic acids instruct cells to produce the desired therapeutic protein internally. This approach underlies modalities such as gene therapy, DNA vaccines, and mRNA-based therapeutics, which have gained wide attention due to their successful application in areas including COVID-19 vaccines and emerging protein drug delivery technologies. The specific function, safety, and therapeutic effects depend entirely on the encoded protein; the nucleic acid itself enables expression and does not act as a therapeutic target or mechanism by which a drug directly acts[5][7][9].
In vivo synthesis of therapeutic proteins following delivery of nucleic acid (DNA/mRNA) encoding protein of interest. Protein acts via its own established mechanism post-translation (e.g., antibody, enzyme, hormone action).
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