WO2011056005A3 - 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 ｓｉＲＮＡ 구조 및 그 용도 - Google Patents

안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 ｓｉＲＮＡ 구조 및 그 용도 Download PDF

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WO2011056005A3
WO2011056005A3 PCT/KR2010/007771 KR2010007771W WO2011056005A3 WO 2011056005 A3 WO2011056005 A3 WO 2011056005A3 KR 2010007771 W KR2010007771 W KR 2010007771W WO 2011056005 A3 WO2011056005 A3 WO 2011056005A3
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sirna
present
sirna molecule
effects caused
antisense strands
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PCT/KR2010/007771
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French (fr)
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WO2011056005A2 (ko
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이동기
퓨자듀아
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성균관대학교산학협력단
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Priority to US13/505,965 priority Critical patent/US9260470B2/en
Publication of WO2011056005A2 publication Critical patent/WO2011056005A2/ko
Publication of WO2011056005A3 publication Critical patent/WO2011056005A3/ko

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Abstract

본 발명은 신규한 siRNA 구조 및 그 용도에 관한 것으로, 보다 상세하게는 안티센스 및 센스로 구성되는 이중가닥의 siRNA 분자로서 상기 siRNA의 안티센스 상에, 특히 5' 말단의 2번째 위치에, 단일 뉴클레오티드의 도입에 의한 단일 뉴클레오티드 벌지(bulge)를 적어도 하나 이상 가지는 것을 특징으로 하는 siRNA 분자 및 이를 이용한 목적 유전자의 발현 억제 방법에 관한 것이다. 본 발명에 따른 siRNA 분자는 우수한 목적 유전자 발현 억제 효율을 보이면서도, 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 타겟 선별성이 향상된 새로운 구조의 siRNA 분자를 제공함으로써, 종래의 siRNA 분자들을 대체하여 유전자 치료 등 siRNA를 기반으로 하는 유전자 발현 억제 기법에 널리 사용될 수 있다.
PCT/KR2010/007771 2009-11-04 2010-11-04 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 ｓｉＲＮＡ 구조 및 그 용도 WO2011056005A2 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US13/505,965 US9260470B2 (en) 2009-11-04 2010-11-04 SiRNA structure for minimizing off-target effects caused by antisense strands, and use thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
KR20090105808 2009-11-04
KR10-2009-0105808 2009-11-04

Publications (2)

Publication Number Publication Date
WO2011056005A2 WO2011056005A2 (ko) 2011-05-12
WO2011056005A3 true WO2011056005A3 (ko) 2011-10-27

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PCT/KR2010/007771 WO2011056005A2 (ko) 2009-11-04 2010-11-04 안티센스 가닥에 의한 오프-타겟 효과를 최소화한 신규한 ｓｉＲＮＡ 구조 및 그 용도

Country Status (3)

Country Link
US (1) US9260470B2 (ko)
KR (1) KR101237036B1 (ko)
WO (1) WO2011056005A2 (ko)

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EP2543372A1 (en) * 2011-07-08 2013-01-09 Helmholtz-Zentrum für Infektionsforschung GmbH Medicament for the treatment of liver cancer
ES2627120T3 (es) * 2011-07-08 2017-07-26 Helmholtz-Zentrum für Infektionsforschung GmbH Medicamento para el tratamiento del cáncer de hígado
CN108148838A (zh) * 2012-05-22 2018-06-12 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
EP2865757A1 (en) 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the PDK1 gene.
EP2865756A1 (en) 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the FLAP gene.
EP2865758A1 (en) 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the ORAI1 gene
WO2015093769A1 (ko) * 2013-12-17 2015-06-25 성균관대학교산학협력단 오프-타겟을 막기 위해 변형된 ｒｎａ 간섭 유도 핵산 및 그 용도
KR101696704B1 (ko) 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
MA44908A (fr) 2015-09-08 2018-07-18 Sylentis Sau Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp
WO2018098352A2 (en) 2016-11-22 2018-05-31 Jun Oishi Targeting kras induced immune checkpoint expression
CA3046963A1 (en) 2016-12-14 2018-06-21 Janssen Biotech, Inc. Cd8a-binding fibronectin type iii domains
KR102321426B1 (ko) 2017-02-21 2021-11-05 올릭스 주식회사 남성형 탈모 표적 유전자의 발현을 억제하는 비대칭 siRNA
JP7162918B2 (ja) * 2018-05-31 2022-10-31 コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション マイクロｒｎａを抑制する変形核酸及びその用途
KR102321425B1 (ko) * 2019-01-18 2021-11-04 올릭스 주식회사 NRL(Neural retina leucine zipper)의 발현을 억제하는 비대칭 siRNA
CN114786682A (zh) 2019-10-14 2022-07-22 Aro生物疗法公司 结合cd71的纤维粘连蛋白iii型结构域
WO2021076574A2 (en) 2019-10-14 2021-04-22 Aro Biotherapeutics Company Fn3 domain-sirna conjugates and uses thereof
KR102259402B1 (ko) * 2020-06-04 2021-06-01 주식회사 아임뉴런바이오사이언스 개선된 안정성을 갖는 핵산 분자 및 이의 용도
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KR20240031947A (ko) * 2021-04-14 2024-03-08 에이알오 바이오테라퓨틱스 컴패니 Fn3 도메인-시르나 접합체 및 이의 용도

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KR20110049733A (ko) 2011-05-12
US20130130377A1 (en) 2013-05-23
US9260470B2 (en) 2016-02-16
KR101237036B1 (ko) 2013-02-25
WO2011056005A2 (ko) 2011-05-12

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