JP6741590B2 - コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 - Google Patents

コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 Download PDF

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JP6741590B2
JP6741590B2 JP2016564200A JP2016564200A JP6741590B2 JP 6741590 B2 JP6741590 B2 JP 6741590B2 JP 2016564200 A JP2016564200 A JP 2016564200A JP 2016564200 A JP2016564200 A JP 2016564200A JP 6741590 B2 JP6741590 B2 JP 6741590B2
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hldlr
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ldlr
aav
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ウイルソン，ジエームス・エム
ラダー，ダニエル・ジエイ
ソマナサン，スリアンラヤン
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A—HUMAN NECESSITIES
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2016564200A 2014-04-25 2015-04-24 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 Active JP6741590B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461984620P 2014-04-25 2014-04-25
US61/984,620 2014-04-25
US201462022627P 2014-07-09 2014-07-09
US62/022,627 2014-07-09
PCT/US2015/027572 WO2015164778A1 (en) 2014-04-25 2015-04-24 Ldlr variants and their use in compositions for reducing cholesterol levels

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JP2020074046A Division JP7280853B2 (ja) 2014-04-25 2020-04-17 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用

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JP2017513504A JP2017513504A (ja) 2017-06-01
JP2017513504A5 JP2017513504A5 (OSRAM) 2018-06-07
JP6741590B2 true JP6741590B2 (ja) 2020-08-19

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JP2016564200A Active JP6741590B2 (ja) 2014-04-25 2015-04-24 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用
JP2020074046A Active JP7280853B2 (ja) 2014-04-25 2020-04-17 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用
JP2023078988A Pending JP2023100960A (ja) 2014-04-25 2023-05-12 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用

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JP2023078988A Pending JP2023100960A (ja) 2014-04-25 2023-05-12 コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用

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US (2) US11555059B2 (OSRAM)
EP (2) EP3919508A1 (OSRAM)
JP (3) JP6741590B2 (OSRAM)
BR (1) BR112016024379A2 (OSRAM)
CA (1) CA2946392A1 (OSRAM)
ES (1) ES2876409T3 (OSRAM)
WO (1) WO2015164778A1 (OSRAM)

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EP3400304B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia

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EP3134431B1 (en) 2021-04-07
JP2017513504A (ja) 2017-06-01
CA2946392A1 (en) 2015-10-29
JP2023100960A (ja) 2023-07-19
EP3919508A1 (en) 2021-12-08
US11555059B2 (en) 2023-01-17
JP7280853B2 (ja) 2023-05-24
US20170101458A1 (en) 2017-04-13
JP2020120673A (ja) 2020-08-13
BR112016024379A2 (pt) 2017-10-10

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