US 8,273,866 B2 · RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)

Abstract

The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

Claims12

Dependent claims

A short interfering RNA (siRNA) molecule having a sense strand and an antisense strand that mediates RNA interference, wherein:

(a) each strand is between 18 and 24 nucleotides in length;
(b) the sense strand comprises 10 or more 2′-deoxy, 2′-O-methyl, 2′-deoxy-2′-fluoro, or universal base modified nucleotides, and a terminal cap molecule at the 3′-end, the 5′-end, or both 3′ and 5′-ends of the sense strand;
(c) the antisense strand comprises 10 or more 2′-deoxy, 2′-O-methyl, 2′-deoxy-2′-fluoro, or universal base modified nucleotides;
and
(d) 10 or more pyrimidine nucleotides of the sense and antisense strand are 2′-deoxy, 2′-O-methyl or 2′-deoxy-2′-fluoro nucleotides.

2 more elements

Dependencies are inferred from the claim text.

Facts

Application

10/720,448Filed 24 Nov 2003 · continuation in part

Priority

20 Feb 2002

Published

US 2009/0137500 A128 May 2009

Granted

25 Sep 2012

Term ends

Not estimatedThe source records don’t support an estimate.

Classification5 codes

C12N 15/113
DNA or RNA fragments; Modified forms thereof ; Non-coding nucleic acids having a biological activity: non-coding nucleic acids modulating the…
C12N 2310/14
Type of nucleic acid: interfering nucleic acids [NA]
C12N 2310/141
Interfering nucleic acids [NA]: microRNAs, miRNAs
C12N 2310/321
Of the sugar: 2'-O-R Modification

Family

Application60/358,580Us provisional application · filed 20 Feb 2002

Application60/363,124Us provisional application · filed 11 Mar 2002

Application60/386,782Us provisional application · filed 6 Jun 2002

Application60/406,784Us provisional application · filed 29 Aug 2002

Application60/408,378Us provisional application · filed 5 Sep 2002

Application60/409,293Us provisional application · filed 9 Sep 2002

Application60/440,129Us provisional application · filed 15 Jan 2003

PCT/US03/05346Parent of this continuation-in-part · filed 20 Feb 2003

PCT/US03/05028Parent of this continuation-in-part · filed 20 Feb 2003

Application10/444,853Parent of this continuation-in-part · filed 23 May 2003

Application10/693,059Parent of this continuation-in-part · filed 23 Oct 2003

US 2009/0137500 A1Pre-grant publication · published 28 May 2009

US 8,202,979 B2Parent of this continuation-in-part

US 2004/0192626 A1Published application

US 2008/0039414 A1Published application

US 8,273,866 B2This patent

From the US filing history. Foreign family members aren’t listed.

RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)

Life of this patent

RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)

Life of this patent

Abstract

Claims12