JP6799058B2 - アレル選択的な遺伝子編集およびその使用 - Google Patents

アレル選択的な遺伝子編集およびその使用 Download PDF

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JP6799058B2
JP6799058B2 JP2018515082A JP2018515082A JP6799058B2 JP 6799058 B2 JP6799058 B2 JP 6799058B2 JP 2018515082 A JP2018515082 A JP 2018515082A JP 2018515082 A JP2018515082 A JP 2018515082A JP 6799058 B2 JP6799058 B2 JP 6799058B2
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nucleotides
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monomer
gene
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JP2018527013A (ja
JP2018527013A5 (enExample
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パドマナブ・チブクラ
グランサム レイチェル・ウィルキー−
グランサム レイチェル・ウィルキー−
キヨシ・タチカワ
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Arcturus Therapeutics Inc
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
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    • A—HUMAN NECESSITIES
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Molecular Biology (AREA)
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JP2018515082A 2015-09-21 2016-09-21 アレル選択的な遺伝子編集およびその使用 Active JP6799058B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562221407P 2015-09-21 2015-09-21
US62/221,407 2015-09-21
US201662330827P 2016-05-02 2016-05-02
US62/330,827 2016-05-02
PCT/US2016/052889 WO2017053431A2 (en) 2015-09-21 2016-09-21 Allele selective gene editing and uses thereof

Publications (3)

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JP2018527013A JP2018527013A (ja) 2018-09-20
JP2018527013A5 JP2018527013A5 (enExample) 2019-10-31
JP6799058B2 true JP6799058B2 (ja) 2020-12-09

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US (1) US10369232B2 (enExample)
EP (1) EP3353298B1 (enExample)
JP (1) JP6799058B2 (enExample)
CN (1) CN108350454B (enExample)
ES (1) ES2959815T3 (enExample)
WO (1) WO2017053431A2 (enExample)

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JP2018527013A (ja) 2018-09-20
ES2959815T3 (es) 2024-02-28
US20170080107A1 (en) 2017-03-23
CN108350454A (zh) 2018-07-31
WO2017053431A2 (en) 2017-03-30
EP3353298A4 (en) 2019-09-25
EP3353298B1 (en) 2023-09-13
EP3353298A2 (en) 2018-08-01
US10369232B2 (en) 2019-08-06
WO2017053431A3 (en) 2017-06-01
CN108350454B (zh) 2022-05-10
CA3002647A1 (en) 2017-03-30

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