CA2804511C - Single-stranded nucleic acid molecule for controlling gene expression - Google Patents

Single-stranded nucleic acid molecule for controlling gene expression Download PDF

Info

Publication number
CA2804511C
CA2804511C CA2804511A CA2804511A CA2804511C CA 2804511 C CA2804511 C CA 2804511C CA 2804511 A CA2804511 A CA 2804511A CA 2804511 A CA2804511 A CA 2804511A CA 2804511 C CA2804511 C CA 2804511C
Authority
CA
Canada
Prior art keywords
region
side region
stranded rna
rna molecule
expression
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2804511A
Other languages
English (en)
French (fr)
Other versions
CA2804511A1 (en
Inventor
Tadaaki Ohgi
Hirotake Hayashi
Hisao SHIROHZU
Tomohiro HAMASAKI
Akihiro Itoh
Hiroshi Suzuki
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Toray Industries Inc
Original Assignee
Bonac Corp
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 Bonac Corp filed Critical Bonac Corp
Publication of CA2804511A1 publication Critical patent/CA2804511A1/en
Application granted granted Critical
Publication of CA2804511C publication Critical patent/CA2804511C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2804511A 2010-07-08 2011-07-08 Single-stranded nucleic acid molecule for controlling gene expression Active CA2804511C (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
JP2010156122 2010-07-08
JP2010-156122 2010-07-08
JP2010-174915 2010-08-03
JP2010174915 2010-08-03
JP2010-230808 2010-10-13
JP2010230808 2010-10-13
JP2010-230806 2010-10-13
JP2010230806 2010-10-13
JP2010269824 2010-12-02
JP2010-269823 2010-12-02
JP2010269823 2010-12-02
JP2010-269824 2010-12-02
PCT/JP2011/065737 WO2012005368A1 (ja) 2010-07-08 2011-07-08 遺伝子発現制御のための一本鎖核酸分子

Publications (2)

Publication Number Publication Date
CA2804511A1 CA2804511A1 (en) 2012-01-12
CA2804511C true CA2804511C (en) 2021-07-06

Family

ID=45441339

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2804511A Active CA2804511C (en) 2010-07-08 2011-07-08 Single-stranded nucleic acid molecule for controlling gene expression

Country Status (17)

Country Link
EP (2) EP2431466B1 (enExample)
JP (3) JP4968811B2 (enExample)
KR (1) KR101849798B1 (enExample)
CN (2) CN104818276B (enExample)
AU (1) AU2011274854B2 (enExample)
BR (1) BR112013000481A2 (enExample)
CA (1) CA2804511C (enExample)
DK (2) DK2431466T3 (enExample)
ES (2) ES2550487T3 (enExample)
HU (2) HUE026520T2 (enExample)
MX (1) MX352568B (enExample)
NZ (2) NZ606232A (enExample)
PT (2) PT2933333T (enExample)
RU (1) RU2628311C2 (enExample)
SG (2) SG196779A1 (enExample)
TW (2) TWI527901B (enExample)
WO (1) WO2012005368A1 (enExample)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8691782B2 (en) 2010-08-03 2014-04-08 Bonac Corporation Single-stranded nucleic acid molecule having nitrogen-containing alicyclic skeleton
WO2013077446A1 (ja) * 2011-11-26 2013-05-30 株式会社ボナック 遺伝子発現制御のための一本鎖核酸分子
WO2013133393A1 (ja) * 2012-03-07 2013-09-12 学校法人東京医科大学 Vegf遺伝子の発現抑制用一本鎖核酸分子
KR20140139512A (ko) * 2012-03-29 2014-12-05 고쿠리쓰다이가쿠호진 규슈다이가쿠 페리오스틴 유전자의 발현 억제 핵산 분자, 페리오스틴 유전자의 발현 억제 방법 및 그 용도
US9663784B2 (en) 2012-05-26 2017-05-30 Bonac Corporation Single-stranded nucleic acid molecule for regulating expression of gene having delivering function
CN105899663B (zh) 2013-12-26 2019-07-26 学校法人东京医科大学 用于基因表达控制的人工模拟miRNA及其用途
CN106068324B (zh) 2013-12-27 2020-12-29 株式会社博纳克 控制基因表达的人工匹配型miRNA及其用途
WO2016098782A1 (ja) 2014-12-15 2016-06-23 株式会社ボナック TGF-β1発現抑制のための一本鎖核酸分子
WO2016104775A1 (ja) 2014-12-27 2016-06-30 株式会社ボナック 遺伝子発現制御のための天然型miRNAおよびその用途
JP6602847B2 (ja) 2015-03-27 2019-11-06 株式会社ボナック デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子
PL3369816T3 (pl) 2015-10-30 2024-07-22 Toray Industries, Inc. KOMPOZYCJA STABILNIE ZAWIERAJĄCA CZĄSTECZKĘ JEDNONICIOWEGO KWASU NUKLEINOWEGO, KTÓRA HAMUJE EKSPRESJĘ GENU TGF-β1
CA3009769C (en) 2015-12-29 2021-01-12 National University Corporation Hokkaido University Single-stranded nucleic acid molecule inhibiting expression of prorenin gene or prorenin receptor gene, and use thereof
KR102255995B1 (ko) 2016-01-30 2021-05-25 가부시키가이샤 보낙 인공 단일 가이드 rna 및 이의 용도
WO2018182008A1 (ja) 2017-03-31 2018-10-04 株式会社ボナック 遺伝子発現制御機能を有する環状型核酸分子
WO2018199338A1 (ja) * 2017-04-27 2018-11-01 国立大学法人広島大学 B型肝炎治療用核酸分子
CN110996969B (zh) 2017-07-28 2023-02-28 杏林制药株式会社 纤维化疾病治疗剂
EP3673066A4 (en) 2017-09-15 2021-05-26 Commonwealth Scientific and Industrial Research Organisation RNA MOLECULES
WO2019074071A1 (ja) 2017-10-11 2019-04-18 日東電工株式会社 核酸分子発現の調節
WO2019074110A1 (ja) 2017-10-13 2019-04-18 株式会社ボナック 一本鎖核酸分子およびその製造方法
TWI830718B (zh) * 2018-02-09 2024-02-01 日商住友化學股份有限公司 核酸分子之製造方法
EP3778914A4 (en) 2018-03-30 2022-01-19 Sumitomo Chemical Company, Ltd. Method for producing single-strand rna
BR112020017769A2 (pt) 2018-03-30 2021-01-05 Toray Industries, Inc. Método e kit para produzir uma molécula de rna de fita simples, e, molécula de oligorna de fita simples.
ES2981806T3 (es) * 2018-10-02 2024-10-10 Toray Industries Método para producir moléculas de ARN monocatenario en horquilla
EP3915567A4 (en) 2019-01-25 2022-09-21 Kyorin Pharmaceutical Co., Ltd. Therapeutic agent for fibrosis
KR20220079832A (ko) 2019-10-11 2022-06-14 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
CN115003682A (zh) 2020-01-29 2022-09-02 住友化学株式会社 核酸寡聚物的制造方法
US12391716B2 (en) 2020-01-29 2025-08-19 Sumitomo Chemical Company, Limited Process of preparing nucleic acid oligomer
JP7613130B2 (ja) * 2020-03-25 2025-01-15 味の素株式会社 リガーゼ変異体
KR20220160010A (ko) 2020-03-27 2022-12-05 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
KR20230034969A (ko) 2020-07-09 2023-03-10 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
KR20230074205A (ko) 2020-09-24 2023-05-26 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
CN117980285A (zh) 2021-09-28 2024-05-03 住友化学株式会社 纯化二氯乙酸的制造方法
CN118891269A (zh) 2022-03-23 2024-11-01 住友化学株式会社 核酸寡聚物的制造方法
JP7201190B1 (ja) 2022-06-03 2023-01-10 株式会社セツロテック 遺伝子改変動物由来の動物繊維
EP4477659A4 (en) 2022-10-27 2025-07-30 Sumitomo Chemical Co METHOD FOR PRODUCING AN OLIGONUCLEOTIDE

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5631148A (en) * 1994-04-22 1997-05-20 Chiron Corporation Ribozymes with product ejection by strand displacement
US6509323B1 (en) 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
FR2790757B1 (fr) * 1999-03-09 2003-08-01 Bioalliance Pharma Oligonucleotides contenant une sequence antisens stabilises par une structure secondaire et compositions pharmaceutiques les contenant.
TWI321054B (en) 2000-12-19 2010-03-01 California Inst Of Techn Compositions containing inclusion complexes
AU2003219900A1 (en) * 2002-02-22 2003-09-09 James R. Eshleman Antigene locks and therapeutic uses thereof
US7452987B2 (en) * 2002-08-05 2008-11-18 Silence Therapeutics Aktiengesellschaft (Ag) Interfering RNA molecules
AU2003256857A1 (en) * 2002-08-08 2004-02-25 Dharmacon, Inc. Short interfering rnas having a hairpin structure containing a non-nucleotide loop
EP1669450B1 (en) * 2003-09-30 2011-11-09 AnGes MG, Inc. Staple type oligonucleotide and drug comprising the same
CA2572151A1 (en) * 2004-06-30 2006-08-24 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a non-phosphate backbone linkage
US20090005332A1 (en) * 2004-12-30 2009-01-01 Hauser Todd M Compositions and Methods for Modulating Gene Expression Using Self-Protected Oligonucleotides
WO2007108621A1 (en) * 2006-03-17 2007-09-27 Bioneer Corporation Sirna for inhibiting nf-kb/rela expression
JP5296328B2 (ja) 2007-05-09 2013-09-25 独立行政法人理化学研究所 1本鎖環状rnaおよびその製造方法
WO2009074076A1 (en) * 2007-11-29 2009-06-18 Suzhou Ribo Life Science Co., Ltd A complex molecule interfering the expression of target genes and its preparing methods
WO2009076321A2 (en) * 2007-12-07 2009-06-18 Halo-Bio Rnai Therapeutics Inc. Compositions and methods for modulating gene expression using asymmetrically-active precursor polynucleotides
EP2256191A4 (en) * 2008-02-15 2011-07-06 Riken CYCLIC SINGLE NUCLEIC ACID COMPLEX AND METHOD FOR THE PRODUCTION THEREOF
JP5187580B2 (ja) 2008-12-26 2013-04-24 丸紅建材リース株式会社 掘削機の泥土飛散防止装置
JP2010174915A (ja) 2009-01-27 2010-08-12 Toyota Motor Corp 高圧ガスタンク
JP5233790B2 (ja) 2009-03-26 2013-07-10 船井電機株式会社 液晶モジュール
JP5449815B2 (ja) 2009-03-26 2014-03-19 住友化学株式会社 偏光板の製造方法
JP2010269823A (ja) 2009-05-21 2010-12-02 British American Tobacco Japan Kk 喫煙品用容器
JP4885255B2 (ja) 2009-05-21 2012-02-29 株式会社エフピコ 包装用容器

Also Published As

Publication number Publication date
CA2804511A1 (en) 2012-01-12
MX2013000315A (es) 2013-05-01
SG196779A1 (en) 2014-02-13
AU2011274854B2 (en) 2015-04-16
EP2431466A1 (en) 2012-03-21
JP4968811B2 (ja) 2012-07-04
ES2550487T3 (es) 2015-11-10
EP2431466B1 (en) 2015-10-07
JP6162182B2 (ja) 2017-07-12
CN104818276A (zh) 2015-08-05
MX352568B (es) 2017-11-29
JP5804642B2 (ja) 2015-11-04
TW201217523A (en) 2012-05-01
JP2012130342A (ja) 2012-07-12
NZ606232A (en) 2015-06-26
TWI527901B (zh) 2016-04-01
CN102918156B (zh) 2015-11-25
RU2013105329A (ru) 2014-08-20
AU2011274854A1 (en) 2013-02-14
CN102918156A (zh) 2013-02-06
EP2431466A4 (en) 2012-04-25
DK2431466T3 (en) 2016-01-11
ES2670017T3 (es) 2018-05-29
CN104818276B (zh) 2020-12-11
KR101849798B1 (ko) 2018-04-17
PT2933333T (pt) 2018-04-24
DK2933333T3 (en) 2018-04-30
EP2933333A1 (en) 2015-10-21
HK1181423A1 (zh) 2013-11-08
WO2012005368A1 (ja) 2012-01-12
EP2933333B1 (en) 2018-02-21
TWI573870B (zh) 2017-03-11
TW201627500A (zh) 2016-08-01
HUE037500T2 (hu) 2018-08-28
JP2015231381A (ja) 2015-12-24
HUE026520T2 (en) 2016-06-28
KR20130090792A (ko) 2013-08-14
SG186899A1 (en) 2013-02-28
NZ704812A (en) 2017-01-27
JPWO2012005368A1 (ja) 2013-09-05
PT2431466E (pt) 2016-01-08
RU2628311C2 (ru) 2017-08-15
BR112013000481A2 (pt) 2019-10-15

Similar Documents

Publication Publication Date Title
CA2804511C (en) Single-stranded nucleic acid molecule for controlling gene expression
EP2628801B1 (en) Single-stranded nucleic acid molecule having nitrogen-containing alicyclic skeleton
US8785121B2 (en) Single-stranded nucleic acid molecule for controlling gene expression
US9206422B2 (en) Single-stranded nucleic acid molecule having nitrogen-containing alicyclic skeleton
EP2801617B1 (en) Single-stranded nucleic acid molecule having amino acid backbone
WO2013077446A1 (ja) 遺伝子発現制御のための一本鎖核酸分子
HK1189910B (en) Single-stranded rna molecule having nitrogen-containing alicyclic skeleton
HK1202310B (en) Single-stranded nucleic acid molecule having amino acid backbone

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20160428