Controlled in-cell activation of RNA therapeutics using bond-cleaving bio-orthogonal chemistry.

ABSTRACT: Temporal control of siRNA activation is a major challenge for RNAi-based therapeutics. The majority of the reported siRNA delivery systems rely on environmental factors, such as differences in extracellular and intracellular redox potential, ATP concentration, or pH to activate an siRNA payload. However dynamic endogenous environments are far too complex to rely on for controllable siRNA release and can result in premature siRNA activation prior to reaching the intended biological target. In addition, there are uncertainties about timing, degree and rate of the siRNA activation with spontaneous release approaches. Herein we describe a bio-orthogonal chemistry approach to address this important challenge. With our approach we were able achieve two major goals: complete siRNA inactivation up

SUBMITTER: Khan I 

PROVIDER: S-EPMC5621156 | biostudies-literature | 2017 Aug

REPOSITORIES: biostudies-literature