RNAi – A Tool for Target Finding in New Drug Development

Virendra   S.   Gomase; Somnath      Tagore

doi:10.2174/138920008783884777

Abstract

RNAi (RNA interference) refers to the introduction of homologous double stranded RNA (dsRNA) to specifically target a genes product, resulting in null or hypomorphic phenotypes. Long double-stranded RNAs (dsRNAs; typically > 200 nt) can be used to silence the expression of target genes in a variety of organisms and cell types (e.g., worms, fruit flies, and plants). The long dsRNAs enter a cellular pathway that is commonly referred to as the RNA interference (RNAi) pathway. RNAi is being considered as an important tool not only for functional genomics, but also for gene-specific therapeutic activities that target the mRNAs of disease-related genes. RNAi plays a very important role in endogenous cellular processes, such as heterochromatin formation, developmental control and serves as an antiviral defense mechanism. RNAi has shown great potential for use as a tool for target finding in new drug development, molecular biological discovery, analysis and therapeutics. RNAi pathway is involved in post-transcription silencing, transcriptional silencing and epigenetic silencing as well as its use as a tool for forward genetics and therapeutics.

Keywords: RNAi, Gene silencing, RNA silencing, Antisense RNA, neurological disorders

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