RNA interference: mechanisms, technical challenges, and therapeutic opportunities

Methods Mol Biol. 2015:1218:1-15. doi: 10.1007/978-1-4939-1538-5_1.

Abstract

The ability to inhibit gene expression via RNA interference (RNAi) has a broad therapeutic potential for various human diseases such as infections and cancers. Recent advances in mechanistic understanding of RNAi have improved the design of functional small interfering (si) RNAs with superior potency and specificity. With respect to delivery, new developments in delivery strategies have facilitated preclinical and clinical siRNA applications. This review provides valuable insights to guide the design and delivery of therapeutic siRNAs.

Publication types

  • Review

MeSH terms

  • Argonaute Proteins / genetics
  • Argonaute Proteins / metabolism
  • Biological Transport
  • Carboxypeptidases / genetics
  • Carboxypeptidases / metabolism
  • Clinical Trials as Topic
  • DEAD-box RNA Helicases / genetics
  • DEAD-box RNA Helicases / metabolism
  • Drug Carriers / chemistry
  • Drug Carriers / metabolism*
  • Exosomes / metabolism
  • Humans
  • Liver Neoplasms / genetics
  • Liver Neoplasms / pathology
  • Liver Neoplasms / therapy*
  • RNA Interference*
  • RNA, Messenger / antagonists & inhibitors
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • RNA, Small Interfering / genetics*
  • RNA, Small Interfering / metabolism
  • Ribonuclease III / genetics
  • Ribonuclease III / metabolism

Substances

  • AGO2 protein, human
  • Argonaute Proteins
  • Drug Carriers
  • RNA, Messenger
  • RNA, Small Interfering
  • DICER1 protein, human
  • DROSHA protein, human
  • Ribonuclease III
  • Carboxypeptidases
  • SCPEP1 protein, human
  • DEAD-box RNA Helicases