CN105308183A - 改变胆固醇水平的组合物和方法 - Google Patents

改变胆固醇水平的组合物和方法 Download PDF

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Publication number
CN105308183A
CN105308183A CN201480028292.8A CN201480028292A CN105308183A CN 105308183 A CN105308183 A CN 105308183A CN 201480028292 A CN201480028292 A CN 201480028292A CN 105308183 A CN105308183 A CN 105308183A
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Prior art keywords
mmrna
polynucleotides
modified
region
sequence
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Pending
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CN201480028292.8A
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Chinese (zh)
Inventor
S·G·霍格
E·艺春·黄
J·B·博伦
贾斯汀·基尔德
安东宁·德富热罗勒
杰夫·L·埃尔斯沃思
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Moderna Inc
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Moderna Therapeutics Inc
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Publication of CN105308183A publication Critical patent/CN105308183A/zh
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    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0073—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen 1.14.13
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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13017—Cholesterol 7-alpha-monooxygenase (1.14.13.17)

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Diabetes (AREA)
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  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CN201480028292.8A 2013-03-15 2014-03-14 改变胆固醇水平的组合物和方法 Pending CN105308183A (zh)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201361786737P 2013-03-15 2013-03-15
US61/786,737 2013-03-15
US201361828214P 2013-05-29 2013-05-29
US61/828,214 2013-05-29
US201361839488P 2013-06-26 2013-06-26
US61/839,488 2013-06-26
US201361903474P 2013-11-13 2013-11-13
US61/903,474 2013-11-13
US14/135,887 US8980864B2 (en) 2013-03-15 2013-12-20 Compositions and methods of altering cholesterol levels
US14/135,887 2013-12-20
PCT/US2014/027453 WO2014152540A1 (en) 2013-03-15 2014-03-14 Compositions and methods of altering cholesterol levels

Publications (1)

Publication Number Publication Date
CN105308183A true CN105308183A (zh) 2016-02-03

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
CN201480028292.8A Pending CN105308183A (zh) 2013-03-15 2014-03-14 改变胆固醇水平的组合物和方法

Country Status (12)

Country Link
US (4) US8980864B2 (https=)
EP (1) EP2970987B1 (https=)
JP (1) JP6396415B2 (https=)
KR (1) KR20160008521A (https=)
CN (1) CN105308183A (https=)
AU (1) AU2014239589A1 (https=)
BR (1) BR112015023386A8 (https=)
CA (1) CA2906092A1 (https=)
MX (1) MX2015012401A (https=)
RU (1) RU2015143930A (https=)
SG (1) SG11201507511SA (https=)
WO (1) WO2014152540A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109666727A (zh) * 2018-12-29 2019-04-23 中国药科大学 一种高活性抑制pcsk9表达的微小rna的用途
CN115957350A (zh) * 2015-12-11 2023-04-14 宾夕法尼亚州大学信托人 用于治疗家族性高胆固醇血症的基因疗法

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