Ontology highlight
ABSTRACT:
SUBMITTER: Kole R
PROVIDER: S-EPMC4743652 | biostudies-literature | 2012 Jan
REPOSITORIES: biostudies-literature
Kole Ryszard R Krainer Adrian R AR Altman Sidney S
Nature reviews. Drug discovery 20120120 2
Here, we discuss three RNA-based therapeutic technologies exploiting various oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have different mechanisms of action and effects. RNA interference and antisense oligonucleotides downregulate gene expression by inducing enzyme-dependent degradation of targeted mRNA. Steric-blocking oligonucleotides block the access of cellular machinery to pre-mRNA and mRNA without degrading the RNA. Through this mechanism, steric-blo ...[more]