siRNA therapeutics: Big potential from small RNAs

R. C.C. Ryther; A. S. Flynt; J. A. Phillips; J. G. Patton

doi:10.1038/sj.gt.3302356

siRNA therapeutics: Big potential from small RNAs

R. C.C. Ryther, A. S. Flynt, J. A. Phillips, J. G. Patton

Research output: Contribution to journal › Review article › peer-review

270 Scopus citations

Abstract

RNA interference (RNAi) is now an umbrella term referring to post-transcriptional gene silencing mediated by either degradation or translation arrest of target RNA. This process is initiated by double-stranded RNA with sequence homology driving specificity. The discovery that 21-23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. While much work remains to optimize delivery and maintain specificity, the therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise.

Original language	English
Pages (from-to)	5-11
Number of pages	7
Journal	Gene therapy
Volume	12
Issue number	1
DOIs	https://doi.org/10.1038/sj.gt.3302356
State	Published - Jan 2005

Keywords

Cancer
Delivery
Dominant
RNAi
siRNA

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abstract = "RNA interference (RNAi) is now an umbrella term referring to post-transcriptional gene silencing mediated by either degradation or translation arrest of target RNA. This process is initiated by double-stranded RNA with sequence homology driving specificity. The discovery that 21-23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. While much work remains to optimize delivery and maintain specificity, the therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise.",

keywords = "Cancer, Delivery, Dominant, RNAi, siRNA",

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note = "Funding Information: This work was supported by the National Institutes of Health (DK035592 and GM62487). RCCR was supported by NIH 5T32 GM0347.",

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AU - Flynt, A. S.

AU - Phillips, J. A.

AU - Patton, J. G.

N1 - Funding Information: This work was supported by the National Institutes of Health (DK035592 and GM62487). RCCR was supported by NIH 5T32 GM0347.

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N2 - RNA interference (RNAi) is now an umbrella term referring to post-transcriptional gene silencing mediated by either degradation or translation arrest of target RNA. This process is initiated by double-stranded RNA with sequence homology driving specificity. The discovery that 21-23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. While much work remains to optimize delivery and maintain specificity, the therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise.

AB - RNA interference (RNAi) is now an umbrella term referring to post-transcriptional gene silencing mediated by either degradation or translation arrest of target RNA. This process is initiated by double-stranded RNA with sequence homology driving specificity. The discovery that 21-23 nucleotide RNA duplexes (small-interfering RNAs, siRNAs) mediate RNAi in mammalian cells opened the door to the therapeutic use of siRNAs. While much work remains to optimize delivery and maintain specificity, the therapeutic advantages of siRNAs for treatment of viral infection, dominant disorders, cancer, and neurological disorders show great promise.

KW - Cancer

KW - Delivery

KW - Dominant

KW - RNAi

KW - siRNA

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JO - Gene therapy

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ER -

siRNA therapeutics: Big potential from small RNAs

Abstract

Keywords

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