CN113692444A - 用于抑制cyp27a1的表达的方法和组合物 - Google Patents

用于抑制cyp27a1的表达的方法和组合物 Download PDF

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Publication number
CN113692444A
CN113692444A CN202080028344.7A CN202080028344A CN113692444A CN 113692444 A CN113692444 A CN 113692444A CN 202080028344 A CN202080028344 A CN 202080028344A CN 113692444 A CN113692444 A CN 113692444A
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oligonucleotide
nucleotides
length
antisense strand
sequence
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Chinese (zh)
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U·萨克森纳
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • 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
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0077—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with a reduced iron-sulfur protein as one donor (1.14.15)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • 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]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • 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
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    • 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
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    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/15—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced iron-sulfur protein as one donor, and incorporation of one atom of oxygen (1.14.15)

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  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN202080028344.7A 2019-02-12 2020-02-07 用于抑制cyp27a1的表达的方法和组合物 Withdrawn CN113692444A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962804410P 2019-02-12 2019-02-12
US62/804410 2019-02-12
PCT/US2020/017129 WO2020167593A1 (en) 2019-02-12 2020-02-07 Methods and compositions for inhibiting expression of cyp27a1

Publications (1)

Publication Number Publication Date
CN113692444A true CN113692444A (zh) 2021-11-23

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CN202080028344.7A Withdrawn CN113692444A (zh) 2019-02-12 2020-02-07 用于抑制cyp27a1的表达的方法和组合物

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US (1) US20220186229A1 (https=)
EP (1) EP3908661A1 (https=)
JP (1) JP2022520653A (https=)
KR (1) KR20210132661A (https=)
CN (1) CN113692444A (https=)
AU (1) AU2020221892A1 (https=)
BR (1) BR112021015651A2 (https=)
CA (1) CA3128059A1 (https=)
CL (2) CL2021002120A1 (https=)
IL (1) IL285367A (https=)
MX (1) MX2021009754A (https=)
SG (1) SG11202108532RA (https=)
WO (1) WO2020167593A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024269205A1 (en) * 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

Family Cites Families (17)

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PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
AU2002213489A1 (en) * 2000-10-13 2002-04-22 Genaissance Pharmaceuticals, Inc. Haplotypes of the cyp27a1 gene
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
JP2005508196A (ja) 2001-11-07 2005-03-31 アプレラ コーポレイション 核酸分析の汎用ヌクレオチド
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
JP2012504389A (ja) * 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ， インコーポレイテッド 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法
US8691971B2 (en) 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
AU2009336191B2 (en) 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
JP2015501155A (ja) 2011-10-25 2015-01-15 アイシス ファーマシューティカルズ， インコーポレーテッド Ｇｃｃｒ発現のアンチセンス調整
EP2928877B1 (en) 2012-12-06 2020-01-22 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
JP2017522046A (ja) 2014-06-06 2017-08-10 ソルスティス バイオロジクス，リミティッド 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物
KR101916583B1 (ko) * 2017-04-20 2018-11-07 가천대학교 산학협력단 칼피르조밉을 유효성분으로 함유하는 담석증의 예방 또는 치료용 약학적 조성물

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

Non-Patent Citations (1)

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Title
DAVID R.RALEIGH等: "Cilia-Associated Oxysterols Activate Smoothened", 《MOLECULAR CELL》, vol. 72, no. 2, pages 27 - 28 *

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MX2021009754A (es) 2021-09-08
IL285367A (en) 2021-09-30
US20220186229A1 (en) 2022-06-16
AU2020221892A1 (en) 2021-08-19
CL2021002120A1 (es) 2022-04-01
SG11202108532RA (en) 2021-09-29
BR112021015651A2 (pt) 2021-10-05
WO2020167593A1 (en) 2020-08-20
CA3128059A1 (en) 2020-08-20
JP2022520653A (ja) 2022-03-31
KR20210132661A (ko) 2021-11-04
CL2023003914A1 (es) 2024-07-12
EP3908661A1 (en) 2021-11-17

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Application publication date: 20211123