CN112055598B - 用于抑制gys2表达的组合物和方法 - Google Patents

用于抑制gys2表达的组合物和方法

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Publication number
CN112055598B
CN112055598B CN201980029587.XA CN201980029587A CN112055598B CN 112055598 B CN112055598 B CN 112055598B CN 201980029587 A CN201980029587 A CN 201980029587A CN 112055598 B CN112055598 B CN 112055598B
Authority
CN
China
Prior art keywords
oligonucleotide
nucleotides
oligonucleotides
gys2
sequence
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.)
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Application number
CN201980029587.XA
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English (en)
Chinese (zh)
Other versions
CN112055598A (zh
Inventor
B·D·布朗
N·珀塞尔
H·T·杜德克
C·赖
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.)
Novo Nordisk AS
Original Assignee
Novo Nordisk AS
Dicerna Pharmaceuticals Inc
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Publication of CN112055598A publication Critical patent/CN112055598A/zh
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Publication of CN112055598B publication Critical patent/CN112055598B/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01001—Phosphorylase (2.4.1.1)
    • 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
    • C12N2310/11—Antisense
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01011—Glycogen(starch) synthase (2.4.1.11)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
CN201980029587.XA 2018-03-02 2019-02-15 用于抑制gys2表达的组合物和方法 Active CN112055598B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862637574P 2018-03-02 2018-03-02
US62/637574 2018-03-02
PCT/US2019/018189 WO2019168687A1 (en) 2018-03-02 2019-02-15 Compositions and methods for inhibiting gys2 expression

Publications (2)

Publication Number Publication Date
CN112055598A CN112055598A (zh) 2020-12-08
CN112055598B true CN112055598B (zh) 2026-04-14

Family

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CN201980029587.XA Active CN112055598B (zh) 2018-03-02 2019-02-15 用于抑制gys2表达的组合物和方法

Country Status (10)

Country Link
US (2) US11572562B2 (enExample)
EP (1) EP3740247A4 (enExample)
JP (1) JP7453921B2 (enExample)
KR (1) KR102804353B1 (enExample)
CN (1) CN112055598B (enExample)
AU (1) AU2019227510B2 (enExample)
CA (1) CA3092092A1 (enExample)
IL (2) IL325191A (enExample)
MX (1) MX2020009072A (enExample)
WO (1) WO2019168687A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3092092A1 (en) 2018-03-02 2019-09-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gys2 expression
AU2020268798A1 (en) * 2019-05-03 2021-11-04 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
WO2022223515A2 (en) * 2021-04-19 2022-10-27 Novo Nordisk A/S Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
AU2024269205A1 (en) * 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity

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US20030004122A1 (en) 1997-11-05 2003-01-02 Leonid Beigelman Nucleotide triphosphates and their incorporation into oligonucleotides
US6436703B1 (en) 2000-03-31 2002-08-20 Hyseq, Inc. Nucleic acids and polypeptides
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US20050119210A1 (en) * 2003-05-20 2005-06-02 Xiaobing Be Compositions and methods for diagnosing and treating cancers
US20060078902A1 (en) * 2004-04-15 2006-04-13 Michaeline Bunting Method and compositions for RNA interference
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
EP2285385A4 (en) * 2008-04-15 2013-01-16 Quark Pharmaceuticals Inc COMPOUNDS BASED ON RNSI TO INHIBIT NRF2
DK2341943T3 (en) 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
KR101728655B1 (ko) * 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
JP2014530602A (ja) 2011-10-05 2014-11-20 プロティバ バイオセラピューティクス インコーポレイテッド アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法
AU2015330670B2 (en) * 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
US10689647B2 (en) 2015-05-08 2020-06-23 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA
EP3416689B1 (en) 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11597926B2 (en) * 2017-12-22 2023-03-07 Roche Innovation Center Copenhagen A/S Thiophosphoramidites
CA3092092A1 (en) 2018-03-02 2019-09-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gys2 expression

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Title
Glycogen controls Caenorhabditis elegans lifespan and resistance to oxidative stress;Ivan Gusarov等;nature communications;20170619;第8卷(第15868期);图3注释、第11页左栏第6段、补充实验数据中表5 *

Also Published As

Publication number Publication date
JP2021517826A (ja) 2021-07-29
CA3092092A1 (en) 2019-09-06
EP3740247A1 (en) 2020-11-25
IL325191A (en) 2026-02-01
MX2020009072A (es) 2020-10-08
KR102804353B1 (ko) 2025-05-13
CN112055598A (zh) 2020-12-08
JP7453921B2 (ja) 2024-03-21
US11572562B2 (en) 2023-02-07
KR20200127211A (ko) 2020-11-10
AU2019227510B2 (en) 2026-02-12
IL277058A (en) 2020-10-29
US20230365974A1 (en) 2023-11-16
WO2019168687A1 (en) 2019-09-06
US12534730B2 (en) 2026-01-27
EP3740247A4 (en) 2022-06-08
US20210062197A1 (en) 2021-03-04

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