Small molecule-switchable RNA platform enables control of therapeutic protein expression

Just like a doctor adjusts the dose of a medication to the patient’s needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important as too much of the protein could be toxic, and too little could result in a small or no therapeutic effect.

Although the principle of therapeutic window has been known for a long time, there has been no strategy to implement it safely, limiting the potential applications of gene therapy…