In vitro-transcribed (IVT) mRNA represents a novel generation of drugs and has attracted much attention. The synthetic mRNA can be engineered to deliver genetic information and express proteins elegantly and transiently. However, there are several inherent challenges of the drug class, such as instability, inefficient in vivo delivery of mRNA and among others. The recent technological advances have provided the basis for a broad range of potential applications. For example, mRNA-based immunotherapy, such as cancer immunotherapy and infectious disease vaccines, have come into the stage of clinical development, and acquired encouraging preliminary results in both animal models and humans. Meanwhile, there are several other emerging novel applications, including protein replacement therapy, regenerative medicine, cell engineering as well as gene editing approach. We present here multiple potential mRNA-based applications.

Applications of mRNA


Cancer cell.

As the main application among mRNA-based therapies, mRNA-immunotherapy has been evaluated in numerous preclinical studies and clinical trials, involving infectious diseases, multiple types of cancer and diabetes. Among them, cancer immunotherapy is the field with the longest exploration history of mRNA-based technologies. Thus far, there are mainly ex vivo and in vivo mRNA-based immunotherapy strategies in various cancers. Besides, due to its superior features, including safe administration, high potency, rapid development and low-cost production, mRNA-based therapy holds the potential to cope with multiple emerging or reemerging infectious diseases.

Protein replacement therapy

mRNA mediated gene expression.

The most obvious application of mRNA-based therapy is protein replacement therapy, and the first report of using synthetic mRNA for supplementing a physiological protein was in 1992. Compared with the traditional administration of protein drugs to the body, the mRNA-based protein replacement therapy is based on the supplementation of desired proteins inside the target cells. Therefore, it is more effective and economical.

Regenerative medicine and cell engineering

Embryonic stem cells.

The ultimate aim of regenerative medicine is to replenish the damaged or lost cells or tissues by restoring or establishing their normal physiological state. mRNA-based therapy is an attractive alternative to conventional approaches, which can effectively deliver functional proteins (e.g., growth factors and transcription factors) to a defect site. At present, there are two specific mRNA-based applications developed in regenerative medicine, including the reprogramming and transdifferentiation of somatic cells as well as mesenchymal stem cells (MSCs) engineering.

Gene editing

Gene editing

Apart from the most obvious application for protein replacement therapy, mRNA technology can also be applied in gene editing for expressing the corresponding nucleases. Creative Biogene is a leading provider in the mRNA-based drug R&D service. In virtue of mRNA therapeutics' advantages, gene editing is a therapeutic tool to achieve a more vigorous development.

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