Synthetic mRNA – Emerging New Class of Gene-based Drugs
Nucleic acid drugs have received much attention due to their therapeutic potential in the fields of gene therapy. Although initial attempts used DNA, and today DNA-based vectors for gene therapy are actively under development, including nonreplicating adenoviruses, adeno-associated viruses, lentiviruses, retroviruses, and others. However, DNA-based gene therapy protocols have sometimes resulted in serious adverse effects in patients, and DNA, regardless of whether it enters the cell by a viral or nonviral route, has the potential of integrating into the host genome, causing insertional mutagenesis and activating oncogenes.
In recent years, a promising alternative to DNA-based vectors, for example, the in vitro transcribed (IVT) messenger RNA (mRNA) coding for desired proteins, has increased in importance. The use of mRNA as a genetic vector is theoretically attractive because mRNA does not integrate into the genome, is immediately available for translation to make protein, and provides a transient signal, a feature that is desirable for some applications. In addition, with the development of synthetic method, the synthetic mRNAs can be sequence optimized to improve translatability into proteins, as well as chemically modified to reduce immunogenicity while retaining its translation efficiency. Synthetic mRNA has been proven effective in numerous applications beneficial for human health such as immunizing patients against cancer and infectious diseases, alleviating diseases by restoring deficient proteins, converting somatic cells to pluripotent stem cells to use in regenerative medicine therapies, and engineering the genome by making specific alterations in DNA.
Fig.1 Principles of antigen-encoding mRNA pharmacology. [1]
Creative Biogene, as one of the global leading biotechnology companies, is able to offer a series of premade mRNA products to satisfy your various downstream applications. With our unparalleled expertise and experience in mRNA synthesis, Creative Biogene is available to provide you with the most affordable and high-quality premade mRNA products to ensure your satisfaction in a timely and professional manner.
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References:
- Sahin U, et al. (2014). mRNA-based therapeutics-developing a new class of drugs. Nature reviews Drug discovery, 13(10), 759-780.
- Kwon H, et al. (2018). Emergence of synthetic mRNA: in vitro synthesis of mRNA and its applications in regenerative medicine. Biomaterials, 156, 172-193.
* For research use only. Not intended for any clinical use.