Smart mRNA molecules on the journey towards patients producing their own therapeutic proteins
Development of novel RNA molecules that enable control over the dose of therapeutic proteins that are produced in patients, which makes gene therapy more efficient
Will patients be able in the future to produce their own medicines? That could be possible by targeting synthetic mRNAs (encoding endogenous proteins, antibodies or other therapeutic proteins), to the cells of patients. Before this becomes reality, some important hurdles must be taken. Researchers from the Massachusetts Institute of Technology (MIT) - in collaboration with researchers from the UGent lab for Gene Therapy, headed by CRIG group leader prof. Niek Sanders - have overcome one important obstacle in this futuristic research. In their recent publication in Nature Chemical Biology, they describe synthetic mRNAs which can be switched on and off using small, non-toxic molecules. As a result, it becomes possible to accurately determine the levels of therapeutic proteins that are produced by a synthetic mRNA and also to decide when the synthesis of these proteins must start or stop in the patient.
This method can also have implications for the battle against cancer. The company that was started by the researchers - Strand Therapeutics - started adapting the method to cancer immunotherapy. Using RNA, the researchers want to develop circuits that selectively stimulate immune cells to attack cancer cells, even if these metastasized to difficult-to-access parts of the body.
- read the full publication in Nature Chemical Biology: "Small-molecule-based regulation of RNA-delivered circuits in mammalian cells"
- MIT news article commenting on the research and its applications