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RNA interference
(RNAi)

Andrew Fire and Craig Mello were awarded 2006 Nobel Prize for Physiology or Medicine for the discovery of RNA interference (RNAi). the Nobel Prize in 2006

RNAi (RNA interference) refers to the introduction of homologous double stranded RNA (dsRNA) to specifically target a gene's product(protein), resulting in null or hypomorphic phenotypes.

The ability of interfering RNA to silence genes was discovered in 1998 by American scientists, Andrew Z. Fire and Craig C. Mello, who shared the 2006 Nobel Prize for Physiology or Medicine for their work. Fire and Mello successfully inhibited the expression of specific genes by introducing short double-stranded RNA (dsRNA) segments into the cells of nematodes (Caenorhabditis elegans).

Gene silencing can be achieved by chemically synthesized RNAi triggering molecules, and RNAi can silence “undruggable” genes(cannot be targeted by small molecules or biologics). It is widely considered one of completely new approaches for drug discovery and development.

RNA interference, RNAi

  • Awarded 2006 Nobel Prize for Physiology or Medicine for the discovery of RNA interference (RNAi)
  • Effective gene silencing through chemically composed double stranded RNA
  • Drug development by silencing disease-causing genes
  • New drug development for “undruggable target” which was unapproachable using low molecular weight
    compound drugs or antibodies in the past, made possible for refractory diseases.
  • “ONPATTRP”, first drug for RNAi to be FDA, approved by FDA and in EU (Alnylam US, Aug. 2018)

  ▲ Unlike small molecules or antibodies, one platform technology can quickly develop novel therapeutics against a variety of diseases