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Gene therapy is an experimental technique using nucleic acids as a therapeutic drug to treat disease, which has held great potential to be a preferred treatment option for many diseases. According to the types of therapeutic nucleic acids, gene therapy is generally divided into DNA-based gene therapy and RNA-based gene therapy. Unlike DNA-based gene therapy that introduces a specific DNA fragment into the genome, RNA-based gene therapy delivers a therapeutic RNA into the cytoplasm of target cells, among which messenger RNA (mRNA)-based therapy has emerged as an attractive method in a wide range of therapeutic applications.
By delivery of a specific synthetic mRNA into the cytoplasm (ribosome) of target cells, mRNA therapy works through the induction of the desired functional proteins to replace defective or missing proteins. Since the first attempt was made in the 1990s, remarkable progress has been achieved in optimizing the stability, immunogenicity, and translatability of mRNA therapy over these years. Currently, mRNA therapy has gained significant potential in the applications of vaccination, protein replacement therapy, and genetic disease treatment.Fig.1 An overview of in vitro transcribed mRNA based therapeutics. (Van Hoecke, 2019)
Although mRNA-based gene therapy is not as well studied as DNA-based gene therapy, the use of mRNA as therapeutics holds many critical advantages. Compared with conventional therapeutic strategies and DNA-based gene therapy, the potential virtues of mRNA-based gene treatment mainly include:
Creative Biolabs is a leading global biologics company established in the United States. Over decades of years' expansion and accumulation, we have won a good reputation in offering high-quality and timesaving biotech products and services to customers all over the world. Supported by advanced technologies and abundant experience, we provide a broad spectrum of mRNA products and services to revolutionize your projects.