WO2010090452A3 - 세포 내 전달능이 증가된 작은 간섭 rna 복합체 - Google Patents

세포 내 전달능이 증가된 작은 간섭 rna 복합체 Download PDF

Info

Publication number
WO2010090452A3
WO2010090452A3 PCT/KR2010/000678 KR2010000678W WO2010090452A3 WO 2010090452 A3 WO2010090452 A3 WO 2010090452A3 KR 2010000678 W KR2010000678 W KR 2010000678W WO 2010090452 A3 WO2010090452 A3 WO 2010090452A3
Authority
WO
WIPO (PCT)
Prior art keywords
sirna
transmission capacity
nucleic acid
complex
functional nucleic
Prior art date
Application number
PCT/KR2010/000678
Other languages
English (en)
French (fr)
Other versions
WO2010090452A2 (ko
Inventor
이동기
장찬일
Original Assignee
성균관대학교산학협력단
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
Application filed by 성균관대학교산학협력단 filed Critical 성균관대학교산학협력단
Priority to US13/146,862 priority Critical patent/US20120016007A1/en
Priority to JP2011547817A priority patent/JP5529895B2/ja
Priority to EP10738736.7A priority patent/EP2395085B1/en
Publication of WO2010090452A2 publication Critical patent/WO2010090452A2/ko
Publication of WO2010090452A3 publication Critical patent/WO2010090452A3/ko
Priority to US16/151,867 priority patent/US20190119672A1/en

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N2310/51—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
    • 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
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

본 발명은 세포 내 전달능이 증가된 다중siRNA 복합체 및 다기능성 핵산구조 복합체에 관한 것이다. 본 발명에 따른 siRNA 복합체 및 다기능성 핵산구조 복합체는 종래 복수의 목적 유전자 발현을 억제하기 위한 shRNA 시스템에 비하여 용이하게 화학적 합성될 수 있는 새로운 구조이면서도, 종래의 siRNA 보다 향상된 효율로 복수의 유전자를 동시에 발현 억제시킬 수 있는 장점이 있다. 또한, 높은 세포 내 전달능을 가질 뿐만 아니라, 비특이적인 항바이러스성 반응을 일으킴없이 특이적으로 목적 유전자들을 발현 억제시킬 수 있는 바, 암이나 바이러스성 감염 등을 치료하기 위한 siRNA 기작 매개 치료제로서 매우 유용하다. 아울러, 본 발명에 따른 다기능성 핵산구조 복합체는, siRNA 이외에 miRNA, antagomiR, 안티센스 올리고뉴클레오티드, 압타머 및 라이보자임(ribozyme)과 같은 기능성 핵산올리고뉴클에오티드를 결합하여 동시에 다양한 기능을 제공할 수 있다.
PCT/KR2010/000678 2009-02-04 2010-02-04 세포 내 전달능이 증가된 작은 간섭 rna 복합체 WO2010090452A2 (ko)

Priority Applications (4)

Application Number Priority Date Filing Date Title
US13/146,862 US20120016007A1 (en) 2009-02-04 2010-02-04 Small interference rna complex with increased intracellular transmission capacity
JP2011547817A JP5529895B2 (ja) 2009-02-04 2010-02-04 細胞内の伝達能が増加した低分子干渉ｒｎａ複合体
EP10738736.7A EP2395085B1 (en) 2009-02-04 2010-02-04 Small interference rna complex with increased intracellular transmission capacity
US16/151,867 US20190119672A1 (en) 2009-02-04 2018-10-04 Small interference rna complex with increased intracellular transmission capacity

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
KR20090009036 2009-02-04
KR10-2009-0009036 2009-02-04

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US13/146,862 A-371-Of-International US20120016007A1 (en) 2009-02-04 2010-02-04 Small interference rna complex with increased intracellular transmission capacity
US16/151,867 Continuation US20190119672A1 (en) 2009-02-04 2018-10-04 Small interference rna complex with increased intracellular transmission capacity

Publications (2)

Publication Number Publication Date
WO2010090452A2 WO2010090452A2 (ko) 2010-08-12
WO2010090452A3 true WO2010090452A3 (ko) 2010-11-25

Family

ID=42542511

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/KR2010/000678 WO2010090452A2 (ko) 2009-02-04 2010-02-04 세포 내 전달능이 증가된 작은 간섭 rna 복합체

Country Status (5)

Country Link
US (2) US20120016007A1 (ko)
EP (1) EP2395085B1 (ko)
JP (1) JP5529895B2 (ko)
KR (1) KR101169373B1 (ko)
WO (1) WO2010090452A2 (ko)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102575252B (zh) 2009-06-01 2016-04-20 光环生物干扰疗法公司 用于多价rna干扰的多核苷酸、组合物及其使用方法
KR101679349B1 (ko) 2010-09-14 2016-11-24 엘지전자 주식회사 이동 단말기, 그 구동 방법 및 식별 모듈 카드
CN103328633B (zh) * 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
US8877722B2 (en) 2011-03-25 2014-11-04 Idera Pharmaceuticals, Inc. Compositions for inhibiting gene expression and uses thereof
EP2756080B1 (en) 2011-09-14 2019-02-20 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
MX2018002090A (es) 2015-08-24 2018-09-12 Halo Bio Rnai Therapeutics Inc Nanoparticulas de polinucleótido para modulación de expresión génica y sus usos.
AU2018215684B2 (en) * 2017-02-06 2024-03-07 Mpeg La, Llc Multimeric oligonucleotides having decreased kidney clearance
WO2020014948A1 (zh) * 2018-07-20 2020-01-23 广州市锐博生物科技有限公司 核酸单元及其聚合核酸与应用
KR20210093851A (ko) * 2018-09-28 2021-07-28 서나오믹스, 인크. 표적과의 상보적 상호 작용을 통해 유전자 발현을 조절하는 다수의 올리고뉴클레오티드로 구성된 다중-표적화 핵산 구조체
WO2020144583A1 (en) * 2019-01-08 2020-07-16 Olix Pharmaceuticals, Inc. Cell-penetrating tripodal interfering rna
CN114175467A (zh) 2019-08-02 2022-03-11 谐波传动系统有限公司 旋转致动器
EP4143320A4 (en) * 2020-04-30 2024-06-19 Mpeg LA, L.l.c. MULTIMER OLIGONUCLEOTIDES WITH SPLIT STRANDS
CN114480377A (zh) * 2022-01-27 2022-05-13 浙江浥眸生物科技有限公司 一种刷状核酸组装体和复合纳米粒子及其应用
CN114748640B (zh) * 2022-05-06 2024-02-09 南方医科大学珠江医院 一种pH响应性的siRNA递送系统

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040147476A1 (en) * 1999-04-21 2004-07-29 Wyeth Methods and compositions for inhibiting the function of polynucleotide sequences
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
US20070254362A1 (en) * 2005-09-02 2007-11-01 Nastech Pharmaceutical Company Inc. COMPOSITIONS AND METHODS EMPLOYING UNIVERSAL-BINDING NUCLEOTIDES FOR TARGETING MULTIPLE GENE VARIANTS WITH A SINGLE siRNA DUPLEX
KR20070114923A (ko) * 2006-05-30 2007-12-05 울산대학교 산학협력단 복수의 표적 ｍＲＮＡ에 적용 가능한 ｓｉＲＮＡ염기서열을 추출하는 방법

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050196781A1 (en) * 2001-05-18 2005-09-08 Sirna Therapeutics, Inc. RNA interference mediated inhibition of STAT3 gene expression using short interfering nucleic acid (siNA)
US20050222066A1 (en) * 2001-05-18 2005-10-06 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7785610B2 (en) * 2001-06-21 2010-08-31 Dynavax Technologies Corporation Chimeric immunomodulatory compounds and methods of using the same—III
US20040147466A1 (en) 2002-01-17 2004-07-29 Barman Shikha P. Nucleic acid delivery formulations
US20040053876A1 (en) * 2002-03-26 2004-03-18 The Regents Of The University Of Michigan siRNAs and uses therof
AU2006232920B2 (en) * 2005-04-06 2011-09-29 Ibc Pharmaceuticals, Inc. Methods for generating stably linked complexes composed of homodimers, homotetramers or dimers of dimers and uses
US20070148246A1 (en) * 2005-08-11 2007-06-28 Dan Luo Nucleic Acid-Based Matrixes
JP5186381B2 (ja) * 2005-11-25 2013-04-17 モロゲン・アーゲー Ｒｎａ干渉による遺伝子発現の特異的抑制のためのｄｎａ構築
WO2007091269A2 (en) * 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
US20080311040A1 (en) * 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
WO2009020344A2 (en) * 2007-08-06 2009-02-12 Postech Acad Ind Found Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same
KR100806088B1 (ko) 2007-08-09 2008-02-21 고려대학교 산학협력단 히알루론산과 폴리에틸렌이민으로 이루어진 양이온성고분자 접합체를 이용한 핵산 전달용 조성물
KR100810034B1 (ko) 2007-08-23 2008-03-05 주식회사 바이오폴리메드 세포 증식 및 이동 억제를 위한 ＮＦ-ＫＢ ｐ105의ｓｉＲＮＡ 및 이를 함유하는 조성물
KR101590586B1 (ko) * 2011-05-30 2016-02-01 성균관대학교산학협력단 표적 유전자 발현 억제 및 면역 반응을 동시에 유발하는 이중가닥의 긴 간섭 ｒｎａ

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040147476A1 (en) * 1999-04-21 2004-07-29 Wyeth Methods and compositions for inhibiting the function of polynucleotide sequences
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
US20070254362A1 (en) * 2005-09-02 2007-11-01 Nastech Pharmaceutical Company Inc. COMPOSITIONS AND METHODS EMPLOYING UNIVERSAL-BINDING NUCLEOTIDES FOR TARGETING MULTIPLE GENE VARIANTS WITH A SINGLE siRNA DUPLEX
KR20070114923A (ko) * 2006-05-30 2007-12-05 울산대학교 산학협력단 복수의 표적 ｍＲＮＡ에 적용 가능한 ｓｉＲＮＡ염기서열을 추출하는 방법

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
AMARSANAA JAZAG ET AL: "Single small-interfering RNA expression vector for silencing multiple transforming growth factor-pathway components", NUCLEIC ACIDS RESEARCH, vol. 33, no. 15, 2005, pages E131, XP008152627 *
CHAN IL CHANG ET AL: "A structure-activity relationship study of siRNAs with structural variations", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 359, no. 4, 2007, pages 997 - 1003, XP022188405 *
See also references of EP2395085A4 *

Also Published As

Publication number Publication date
EP2395085A9 (en) 2012-02-29
WO2010090452A2 (ko) 2010-08-12
US20190119672A1 (en) 2019-04-25
EP2395085A2 (en) 2011-12-14
KR20100089796A (ko) 2010-08-12
EP2395085B1 (en) 2015-06-10
JP5529895B2 (ja) 2014-06-25
JP2012516683A (ja) 2012-07-26
US20120016007A1 (en) 2012-01-19
EP2395085A4 (en) 2013-01-23
KR101169373B1 (ko) 2012-07-30

Similar Documents

Publication Publication Date Title
WO2010090452A3 (ko) 세포 내 전달능이 증가된 작은 간섭 rna 복합체
Pasternak et al. Unlocked nucleic acid–an RNA modification with broad potential
WO2003070897A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF TNF AND TNF RECEPTOR SUPERFAMILY GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP2017079776A5 (ko)
WO2004083430A3 (en) SHORT INTERFERING RNA (siRNA) ANALOGUES
WO2013192576A3 (en) Oligonucleotide-based inhibitors comprising locked nucleic acid motif
WO2008061537A3 (en) Oligonucleotides for modulating target rna activity
WO2003072705A3 (en) Rna interference mediated inhibition of cyclin d1 gene expression using short interfering nucleic acid (sina)
WO2012053741A3 (ko) Rna 간섭을 유도하는 핵산 분자 및 그 용도
WO2005045034A3 (en) RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
WO2008109460A3 (en) Nucleic acid compounds for inhibiting ctnnb1 gene expression and uses thereof
WO2012024170A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS B VIRUS (HBV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2003072704A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET DERIVED GROWTH FACTOR (PDGF) AND PLATELET DERIVED GROWTH FACTOR RECEPTOR (PDGFR) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2012018754A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2003070884A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF MDR P-GLYCOPROTEIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
Aaldering et al. Smart functional nucleic acid chimeras: enabling tissue specific RNA targeting therapy
AU2014219019A8 (en) Short interfering nucleic acid (siNA) molecules containing a 2' internucleoside linkage
WO2008094516A3 (en) Multi-targeting short interfering rnas
WO2012178033A3 (en) Serpina1 sirnas: compositions of matter and methods of treatment
WO2010111503A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF THE HIGH AFFINITY IGE RECEPTOR ALPHA CHAIN (FCεRLα) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
WO2010017319A3 (en) Nucleic acid compounds for inhibiting plk1 gene expression and uses thereof
WO2010006973A3 (en) Compositions and methods for inhibiting expression of tgf-beta receptor genes
WO2011081415A3 (ko) c-Met의 발현을 저해하는 siRNA 및 이를 포함하는 항암 조성물
WO2003070887A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF POLYCOMB GROUP PROTEIN EZH2 GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2005007855A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF B7-H1 GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 10738736

Country of ref document: EP

Kind code of ref document: A2

WWE Wipo information: entry into national phase

Ref document number: 2010738736

Country of ref document: EP

WWE Wipo information: entry into national phase

Ref document number: 2011547817

Country of ref document: JP

NENP Non-entry into the national phase

Ref country code: DE

WWE Wipo information: entry into national phase

Ref document number: 13146862

Country of ref document: US