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

Apr 10, 2023

By Okinawa Institute of Science and Technology

Turning genes on and off as easily and predictably as flicking a switch could be a powerful tool in medicine and biotech. A type of technology called a riboswitch might be the key.

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.

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