Title:
NOVEL SIRNA STRUCTURE FOR MINIMIZING OFF-TARGET EFFECTS CAUSED BY ANTISENSE STRANDS, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2011/056005
Kind Code:
A2
Abstract:
The present invention relates to a novel siRNA structure and to the use thereof, and more particularly, to a siRNA molecule and to a target gene silencing method using same, wherein the siRNA molecule is a double-stranded siRNA molecule comprising an antisense strand and a sense strand, and at least one or more single nucleotide bulges induced by the introduction of single nucleotides are present on the antisense strand of the siRNA, specifically at the second position of the 5' end. The present invention provides a siRNA molecule having a novel structure, which exhibits superior efficiency of target gene silencing, minimizes off-target effects caused by antisense strands, and thus has improved target selectivity. Therefore, the siRNA molecule of the present invention can be widely used in siRNA-based gene silencing technologies such as a gene therapy or the like in lieu of conventional siRNA molecules.
Inventors:
LEE DONG KI (KR)
POOJA DUA (KR)
POOJA DUA (KR)
Application Number:
PCT/KR2010/007771
Publication Date:
May 12, 2011
Filing Date:
November 04, 2010
Export Citation:
Assignee:
UNIV SUNGKYUNKWAN FOUND (KR)
LEE DONG KI (KR)
POOJA DUA (KR)
LEE DONG KI (KR)
POOJA DUA (KR)
International Classes:
C12N15/11; A61K48/00; C07H21/02
Foreign References:
| US20050020521A1 | 2005-01-27 |
Other References:
DOENCH, J. G. ET AL.: 'siRNAs can function as miRNAs.' GENES DEV. vol. 17, 15 February 2003, pages 438 - 442
SAXENA, S. ET AL.: 'Small RNAs with Imperfect Match to Endogenous mRNA Repress Translation.' THE JOURNAL OF BIOLOGICAL CHEMISTRY vol. 278, no. 45, 02 September 2003, pages 44312 - 44319
OHRT, T. ET AL.: 'Fluorescence correlation spectroscopy and fluorescence cross-correlation spectroscopy reveal the cytoplasmic origination of loaded nuclear RISC in vivo in human cells.' NUCLEIC ACIDS RESEARCH vol. 36, 08 October 2008, pages 6439 - 6449
MARTIN, S. E. ET AL.: 'Mismatched siRNAs downregulate mRNAs as a function of target site location.' FEBS LETTERS. vol. 580, 05 June 2006, pages 3694 - 3698
SAXENA, S. ET AL.: 'Small RNAs with Imperfect Match to Endogenous mRNA Repress Translation.' THE JOURNAL OF BIOLOGICAL CHEMISTRY vol. 278, no. 45, 02 September 2003, pages 44312 - 44319
OHRT, T. ET AL.: 'Fluorescence correlation spectroscopy and fluorescence cross-correlation spectroscopy reveal the cytoplasmic origination of loaded nuclear RISC in vivo in human cells.' NUCLEIC ACIDS RESEARCH vol. 36, 08 October 2008, pages 6439 - 6449
MARTIN, S. E. ET AL.: 'Mismatched siRNAs downregulate mRNAs as a function of target site location.' FEBS LETTERS. vol. 580, 05 June 2006, pages 3694 - 3698
Attorney, Agent or Firm:
LEE, Cheo Young (KR)
이처영 (KR)
이처영 (KR)
Download PDF: