Knockdown of β-catenin with dicer-substrate siRNAs reduces liver tumor burden in vivo.

ABSTRACT: Despite progress in identifying molecular drivers of cancer, it has been difficult to translate this knowledge into new therapies, because many of the causal proteins cannot be inhibited by conventional small molecule therapeutics. RNA interference (RNAi), which uses small RNAs to inhibit gene expression, provides a promising alternative to reach traditionally undruggable protein targets by shutting off their expression at the messenger RNA (mRNA) level. Challenges for realizing the potential of RNAi have included identifying the appropriate genes to target and achieving sufficient knockdown in tumors. We have developed high-potency Dicer-substrate short-interfering RNAs (DsiRNAs) targeting β-catenin and delivered these in vivo using lipid nanoparticles, resulting in significant reduction

SUBMITTER: Dudek H 

PROVIDER: S-EPMC3978813 | biostudies-literature | 2014 Jan

REPOSITORIES: biostudies-literature