Ontology highlight
ABSTRACT:
SUBMITTER: Boudreau RL
PROVIDER: S-EPMC3242660 | biostudies-literature | 2011 Dec
REPOSITORIES: biostudies-literature
Boudreau Ryan L RL Spengler Ryan M RM Davidson Beverly L BL
Molecular therapy : the journal of the American Society of Gene Therapy 20110927 12
RNA interference (RNAi) provides an approach for the treatment of many human diseases. However, the safety of RNAi-based therapies can be hampered by the ability of small inhibitory RNAs (siRNAs) to bind to unintended mRNAs and reduce their expression, an effect known as off-target gene silencing. Off-targeting primarily occurs when the seed region (nucleotides 2-8 of the small RNA) pairs with sequences in 3'-UTRs of unintended mRNAs and directs translational repression and destabilization of th ...[more]