Rational RNA Therapeutics
“The biggest obstacle to creativity is breaking through the barrier of disbelief.”—Rodney Mullen
Recent clinical successes have demonstrated that small interfering RNAs (siRNAs) can be used to effectively treat transthyretin-related amyloidosis. This breakthrough is likely only the very beginning for siRNA therapeutics--because they exploit the versatility and efficiency of Argonaute2, therapeutic siRNAs could be used to treat or prevent almost any human disease. We are using our intimate knowledge of Argonaute structure and mechanism to devise innovative ways of designing and delivering therapeutic siRNAs into human cells.