RNAi as an experimental and therapeutic tool to study and regulate physiological and disease processes

Christopher P. Dillon, Peter Sandy, Alessio Nencioni, Stephan Kissler, Douglas A. Rubinson, Luk Van Parijs

Research output: Contribution to journalArticlepeer-review

Abstract

Over the past four years RNA interference (RNAi) has exploded onto the research scene as a new approach to manipulate gene expression in mammalian systems. More recently, RNAi has garnered much interest as a potential therapeutic strategy. In this review, we briefly summarize the current understanding of RNAi biology and examine how RNAi has been used to study the genetic basis of physiological and disease processes in mammalian systems. We also explore some of the new developments in the use of RNAi for disease therapy and highlight the key challenges that currently limit its application in the laboratory, as well as in the clinical setting.

Original languageEnglish
Pages (from-to)147-173
Number of pages27
JournalAnnual Review of Physiology
Volume67
DOIs
Publication statusPublished - 2005

Keywords

  • MicroRNA
  • shRNA
  • siRNA
  • Therapy
  • Vector

ASJC Scopus subject areas

  • Physiology

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