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RNA interference and potential therapeutic applications of short interfering RNAs.

Abstract：

:RNA interference is an endogenous gene-silencing mechanism that involves double-stranded RNA-mediated sequence-specific mRNA degradation. The discovery of this pathway together with the elucidation of the structure and function of short interfering RNAs--the effector molecules of RNA interference--has had an enormous impact on experimental biology. RNA interference technologies are currently the most widely utilized techniques in functional genomic studies. Furthermore, there is an intense research effort aimed at developing short interfering RNAs for therapeutic purposes. A number of proof-of-principle experiments have demonstrated the clinical potential of appropriately designed short interfering RNAs in various diseases including viral infections, cancer and neurodegenerative disorders. Already, in such a short time from their discovery, Acuity Pharmaceuticals (August 2004) and Sirna Therapeutics (September 2004) have filed Investigational New Drug applications with the US FDA to begin clinical trials with modified siRNA molecules in patients with age-related macular degeneration. This review will give a brief overview of the mechanism of RNA interference and applications of the pathway in experimental biology will be discussed. The article will focus on recent developments related to the use of RNA interference technologies in mammalian systems and on potential clinical applications of short interfering RNA-mediated RNA interference.

journal_name

Cancer Gene Ther

journal_title

Cancer gene therapy

authors

Karagiannis TC,El-Osta A

doi

10.1038/sj.cgt.7700857

subject

Has Abstract

pub_date

2005-10-01 00:00:00

pages

787-95

issue

10

eissn

0929-1903

issn

1476-5500

pii

7700857

journal_volume

12

pub_type

杂志文章,评审

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