JP2020510648A - 家族性高コレステロール血症を処置するための遺伝子治療 - Google Patents

家族性高コレステロール血症を処置するための遺伝子治療 Download PDF

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JP2020510648A
JP2020510648A JP2019545363A JP2019545363A JP2020510648A JP 2020510648 A JP2020510648 A JP 2020510648A JP 2019545363 A JP2019545363 A JP 2019545363A JP 2019545363 A JP2019545363 A JP 2019545363A JP 2020510648 A JP2020510648 A JP 2020510648A
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raav
vector
aav8
hldlr
dose
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JP2020510648A5 (enExample
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ウイルソン，ジェームス・エム
レイダー，ダニエル・ジェイ
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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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    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61K35/76—Viruses; Subviral particles; Bacteriophages
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • A—HUMAN NECESSITIES
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/035—Animal model for multifactorial diseases
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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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  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
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JP2019545363A 2017-02-20 2018-02-20 家族性高コレステロール血症を処置するための遺伝子治療 Pending JP2020510648A (ja)

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US201762461015P 2017-02-20 2017-02-20
US62/461,015 2017-02-20
PCT/US2018/018678 WO2018152485A1 (en) 2017-02-20 2018-02-20 Gene therapy for treating familial hypercholesterolemia

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US (2) US11554147B2 (enExample)
EP (1) EP3595688A4 (enExample)
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KR (1) KR20190118163A (enExample)
AU (1) AU2018220212A1 (enExample)
CA (1) CA3051011A1 (enExample)
IL (1) IL268276A (enExample)
WO (1) WO2018152485A1 (enExample)

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US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
WO2021080975A1 (en) * 2019-10-21 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing cholesterol levels
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WO2023081807A1 (en) * 2021-11-04 2023-05-11 The Trustees of the University of Pennsylvania Penn Center for Innovation Compositions and methods for reducing pcsk9 levels in a subject

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HUMAN GENE THERAPY CLINICAL DEVELOPMENT, 2013, VOL.24, PP.154-160, JPN6022001760, ISSN: 0005073195 *
HUMAN GENE THERAPY, 2013, VOL.24, PP.19-26, JPN6022001759, ISSN: 0005073196 *
MOLECULAR THERAPY, 2013, VOL.21, NO.2, PP.318-323, JPN6022001758, ISSN: 0005073197 *

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CA3051011A1 (en) 2018-08-23
US20230190837A1 (en) 2023-06-22
EP3595688A4 (en) 2020-12-30
IL268276A (en) 2019-09-26
WO2018152485A1 (en) 2018-08-23
US20200230184A1 (en) 2020-07-23
JP2023071829A (ja) 2023-05-23

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