WO2009020344A3 - Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same - Google Patents

Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same Download PDF

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Publication number
WO2009020344A3
WO2009020344A3 PCT/KR2008/004563 KR2008004563W WO2009020344A3 WO 2009020344 A3 WO2009020344 A3 WO 2009020344A3 KR 2008004563 W KR2008004563 W KR 2008004563W WO 2009020344 A3 WO2009020344 A3 WO 2009020344A3
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WO
WIPO (PCT)
Prior art keywords
sirnas
target genes
preparing
same
multiple target
Prior art date
Application number
PCT/KR2008/004563
Other languages
French (fr)
Other versions
WO2009020344A2 (en
Inventor
Dong Ki Lee
Chan Il Chang
Original Assignee
Dong Ki Lee
Chan Il Chang
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from KR1020070078785A external-priority patent/KR20090014676A/en
Priority claimed from KR1020070078782A external-priority patent/KR100929699B1/en
Priority claimed from KR1020070078783A external-priority patent/KR100929700B1/en
Application filed by Dong Ki Lee, Chan Il Chang filed Critical Dong Ki Lee
Publication of WO2009020344A2 publication Critical patent/WO2009020344A2/en
Publication of WO2009020344A3 publication Critical patent/WO2009020344A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/52—Physical structure branched

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Disclosed are siRNAs shorter than 19 base pairs or nucleotides in length, multi- silencing siRNA, composed of plural siRNAs, capable of regulating the expression of respective target genes, and therapeutics for the treatment of gene control -related diseases, comprising the multi -silencing siRNA as an active ingredient. The siRNAs with fewer nucleotides can be produced at lower cost and can regulate gene expression at a higher efficiency per weight. Also, the multi -silencing siRNAs prepared from the siRNAs are easy to clinically apply, for example, are easily introduced into cells. Thus, the siRNAs find a broad spectrum of applications in the medical industry, including therapeutics for diseases, such as cancers, viral diseases, etc.
PCT/KR2008/004563 2007-08-06 2008-08-06 Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same WO2009020344A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
KR1020070078785A KR20090014676A (en) 2007-08-06 2007-08-06 Small interfering rna (sirna) having a shortened nucleotide sequence
KR10-2007-0078782 2007-08-06
KR1020070078782A KR100929699B1 (en) 2007-08-06 2007-08-06 Interference-induced RNAs that control multiple target genes and methods of making them
KR1020070078783A KR100929700B1 (en) 2007-08-06 2007-08-06 Some single stranded multiple interference induction RNA
KR10-2007-0078783 2007-08-06
KR10-2007-0078785 2007-08-06

Publications (2)

Publication Number Publication Date
WO2009020344A2 WO2009020344A2 (en) 2009-02-12
WO2009020344A3 true WO2009020344A3 (en) 2009-04-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/KR2008/004563 WO2009020344A2 (en) 2007-08-06 2008-08-06 Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same

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WO (1) WO2009020344A2 (en)

Families Citing this family (24)

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AU2009293658A1 (en) 2008-09-22 2010-03-25 James Cardia Reduced size self-delivering RNAi compounds
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010090452A2 (en) * 2009-02-04 2010-08-12 성균관대학교산학협력단 Small interference rna complex with increased intracellular transmission capacity
CN105131067B (en) 2010-03-24 2019-02-19 雷克西制药公司 RNA in skin and fibrotic conditions is interfered
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
CN106074591B (en) 2010-03-24 2020-01-14 菲奥医药公司 RNA interference in ocular symptoms
CA2818662C (en) 2010-10-22 2021-07-06 Sungkyunkwan University Foundation For Corporate Collaboration Nucleic acid molecule inducing rna interference, and uses thereof
DE102011009470A1 (en) * 2011-01-21 2012-08-09 Friedrich-Schiller-Universität Jena Biologically active nucleotide molecules for the targeted killing of cells, use thereof and application kit
JP6139671B2 (en) 2012-05-22 2017-05-31 オリックス ファーマシューティカルズ インコーポレイテッドＯｌｉｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃ． Nucleic acid molecule that induces RNA interference with intracellular penetration ability and use thereof
US9988627B2 (en) 2013-10-04 2018-06-05 Novartis Ag Formats for organic compounds for use in RNA interference
WO2015051044A2 (en) * 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
US10934550B2 (en) 2013-12-02 2021-03-02 Phio Pharmaceuticals Corp. Immunotherapy of cancer
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
EP3188799B1 (en) 2014-09-05 2022-07-06 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting tyr or mmp1
WO2017007825A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
JP6983752B2 (en) 2015-07-06 2021-12-17 フィオ ファーマシューティカルズ コーポレーションPhio Pharmaceuticals Corp. Nucleic acid molecule targeting superoxide dismutase 1 (SOD1)
JP2018531037A (en) 2015-10-19 2018-10-25 アールエックスアイ ファーマシューティカルズ コーポレーション Reduced size self-delivering nucleic acid compounds targeting long non-coding RNAs
JP7027311B2 (en) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ，インコーポレーテッド Treatment of age-related macular degeneration with RNA complexes targeting MyD88 or TLR3
JP7003044B2 (en) 2016-02-02 2022-01-20 オリックス ファーマシューティカルズ，インコーポレーテッド Treatment of angiogenesis-related diseases with RNA complexes targeting ANGPT2 and PDGFB
EP3411480A4 (en) 2016-02-02 2020-01-22 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4R , TRPA1, OR F2RL1
US10301628B2 (en) 2016-04-11 2019-05-28 Olix Pharmaceuticals, Inc. Treatment of idiopathic pulmonary fibrosis using RNA complexes that target connective tissue growth factor
KR101916652B1 (en) 2016-06-29 2018-11-08 올릭스 주식회사 Compounds improving RNA interference of small interfering RNA and use thereof
US11591600B2 (en) 2017-02-10 2023-02-28 OliX Pharmaceuticals. Inc. Long double-stranded RNA for RNA interference

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000063364A2 (en) * 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
WO2004083430A2 (en) * 2003-03-21 2004-09-30 Santaris Pharma A/S SHORT INTERFERING RNA (siRNA) ANALOGUES
US20060178327A1 (en) * 2003-05-30 2006-08-10 Yeung Wah Hin A Inhibition of gene expression by delivery of specially selected double stranded or other forms of small interfering RNA precursors enabling the formation and function of small interfering RNA in vivo and in vitro

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000063364A2 (en) * 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
WO2004083430A2 (en) * 2003-03-21 2004-09-30 Santaris Pharma A/S SHORT INTERFERING RNA (siRNA) ANALOGUES
US20060178327A1 (en) * 2003-05-30 2006-08-10 Yeung Wah Hin A Inhibition of gene expression by delivery of specially selected double stranded or other forms of small interfering RNA precursors enabling the formation and function of small interfering RNA in vivo and in vitro

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
AMARSANAA JAZAG ET AL., NUCLEIC ACIDS RESEARCH, vol. 33, no. 15, 19 August 2005 (2005-08-19), pages 1 - 9 *

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