JP7023108B2 - 組み換えａａｖバリアントおよびその使用 - Google Patents

組み換えａａｖバリアントおよびその使用 Download PDF

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JP7023108B2
JP7023108B2 JP2017521515A JP2017521515A JP7023108B2 JP 7023108 B2 JP7023108 B2 JP 7023108B2 JP 2017521515 A JP2017521515 A JP 2017521515A JP 2017521515 A JP2017521515 A JP 2017521515A JP 7023108 B2 JP7023108 B2 JP 7023108B2
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hsa
mir
aav
raav
disease
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JP2017532966A5 (cg-RX-API-DMAC7.html
JP2017532966A (ja
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ガオ，グアンピン
シエ，ジュン
フロット，テレンス
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ユニバーシティ オブ マサチューセッツ
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    • A—HUMAN NECESSITIES
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
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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
    • C12N15/63—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
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    • 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
    • A61K48/0066—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
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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
    • C12N15/63—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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • A—HUMAN NECESSITIES
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A—HUMAN NECESSITIES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
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    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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JP2017521515A 2014-10-21 2015-10-21 組み換えａａｖバリアントおよびその使用 Active JP7023108B2 (ja)

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US201462066856P 2014-10-21 2014-10-21
US62/066,856 2014-10-21
PCT/US2015/056659 WO2016065001A1 (en) 2014-10-21 2015-10-21 Recombinant aav variants and uses thereof

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EP (1) EP3209311B1 (cg-RX-API-DMAC7.html)
JP (2) JP7023108B2 (cg-RX-API-DMAC7.html)
CN (1) CN107073051B (cg-RX-API-DMAC7.html)
AU (2) AU2015335923B2 (cg-RX-API-DMAC7.html)
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JP2021010372A (ja) * 2014-10-21 2021-02-04 ユニバーシティ オブ マサチューセッツ 組み換えａａｖバリアントおよびその使用
US11834474B2 (en) 2009-05-28 2023-12-05 University Of Massachusetts AAV's and uses thereof

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WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
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CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
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