KR102804353B1 - Gys2 발현을 억제하기 위한 조성물 및 방법 - Google Patents

Gys2 발현을 억제하기 위한 조성물 및 방법 Download PDF

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KR102804353B1
KR102804353B1 KR1020207027734A KR20207027734A KR102804353B1 KR 102804353 B1 KR102804353 B1 KR 102804353B1 KR 1020207027734 A KR1020207027734 A KR 1020207027734A KR 20207027734 A KR20207027734 A KR 20207027734A KR 102804353 B1 KR102804353 B1 KR 102804353B1
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artificial sequence
synthetic polynucleotide
rna
oligonucleotide
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KR20200127211A (ko
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밥 디. 브라운
나탈리 퍼셀
헨릭 티. 두덱
쳉 라이
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노보 노르디스크 에이/에스
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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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
    • C12N15/113—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
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    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01001—Phosphorylase (2.4.1.1)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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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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    • 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
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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
    • 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)

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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
KR1020207027734A 2018-03-02 2019-02-15 Gys2 발현을 억제하기 위한 조성물 및 방법 Active KR102804353B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862637574P 2018-03-02 2018-03-02
US62/637,574 2018-03-02
PCT/US2019/018189 WO2019168687A1 (en) 2018-03-02 2019-02-15 Compositions and methods for inhibiting gys2 expression

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KR20200127211A KR20200127211A (ko) 2020-11-10
KR102804353B1 true KR102804353B1 (ko) 2025-05-13

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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)

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Publication number Priority date Publication date Assignee Title
MX2020009072A (es) 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
IL307154A (en) * 2021-04-19 2023-11-01 Novo Nordisk As Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
JP2026520116A (ja) * 2023-05-11 2026-06-22 大睿生物 ＭＡＳＰ２遺伝子活性を制御するためのｄｓＲＮＡ分子
WO2026162643A1 (en) * 2025-01-31 2026-08-06 Novo Nordisk A/S Nucleic acid constructs for inhibiting gys2 expression

Citations (3)

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WO2001075067A2 (en) 2000-03-31 2001-10-11 Hyseq, Inc. Novel nucleic acids and polypeptides
WO2010105372A1 (en) 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

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US20030004122A1 (en) * 1997-11-05 2003-01-02 Leonid Beigelman Nucleotide triphosphates and their incorporation into oligonucleotides
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional 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
CA2566286A1 (en) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
WO2009144704A2 (en) * 2008-04-15 2009-12-03 Quark Pharmaceuticals, Inc. siRNA COMPOUNDS FOR INHIBITING NRF2
WO2010033225A2 (en) 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
KR20110110776A (ko) * 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
CA2850792A1 (en) 2011-10-05 2013-04-11 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
US10351854B2 (en) * 2014-10-10 2019-07-16 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
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
WO2017141109A1 (en) * 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
CN111448316B (zh) * 2017-12-22 2025-02-14 罗氏创新中心哥本哈根有限公司 新的硫代亚磷酰胺
MX2020009072A (es) 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001075067A2 (en) 2000-03-31 2001-10-11 Hyseq, Inc. Novel nucleic acids and polypeptides
WO2010105372A1 (en) 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Nature Communications, Vol. 8, pp.15868(1-12) (2017)*

Also Published As

Publication number Publication date
EP3740247A1 (en) 2020-11-25
CN112055598A (zh) 2020-12-08
MX2020009072A (es) 2020-10-08
JP2021517826A (ja) 2021-07-29
US20230365974A1 (en) 2023-11-16
US20210062197A1 (en) 2021-03-04
IL277058A (en) 2020-10-29
AU2019227510B2 (en) 2026-02-12
WO2019168687A1 (en) 2019-09-06
CA3092092A1 (en) 2019-09-06
US11572562B2 (en) 2023-02-07
JP7453921B2 (ja) 2024-03-21
KR20200127211A (ko) 2020-11-10
IL325191A (en) 2026-02-01
US12534730B2 (en) 2026-01-27
CN112055598B (zh) 2026-04-14
EP3740247A4 (en) 2022-06-08

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