RATIONAL RNA THERAPEUTICS
The biggest obstacle to creativity is breaking through the barrier of disbelief.
Success in the clinic has demonstrated that small interfering RNAs (siRNAs) can be used to effectively treat transthyretin-related amyloidosis. This breakthrough has ushered in the era of siRNA therapeutics. Because they exploit the versatility and efficiency of Argonaute2, therapeutic siRNAs could be used to regulate essentially any human gene. We are using our intimate knowledge of Argonaute structure and mechanism to devise innovative ways of designing novel siRNAs and controlling Argonaute function.