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New RNA-based toolkit to regulate gene activity could advance regenerative medicine, gene therapy and biotechnology

The Okinawa Institute of Science and Technology (OIST), in collaboration with Astellas Pharma Inc., has developed a new toolkit that uses small molecules to control the activity of a piece of synthetic RNA, and ultimately regulate gene expression. The technology, which was described in the Journal of the American Chemical Society on March 30, 2023, worked in mammalian cell cultures and in mice.

The ability to precisely control whether a gene is turned on or off is expected to lead to more efficient production of compounds that are made using animal cells, and make gene therapy, cell therapy and regenerative medicine safer.

For genes to be expressed, cells make many RNA copies of a section of DNA. These RNA copies, called transcripts, are then used to make the protein. Scientists can take advantage of this process by introducing additional genes (either as DNA or RNA) into cells, which can then be used to make new proteins for a wide variety of applications. For example, in gene therapy, disorders caused by faulty proteins can be treated by introducing genes into body cells that code for working versions of that protein and in regenerative medicine, the expression of inserted genes could reprogram cells. Proteins with drug applications, like hormones and antibodies, can also be easily produced and collected by inserting genes into cultured cells.

However, after introducing DNA or RNA into cells, scientists need to be able to precisely control the production of the protein, so that it is made at the right time and in the right amount. This maximizes productivity and could help avoid unnecessary side effects in patients.

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