JP2018520149A - Ｃｒｉｓｐｒ／ｃａｓ９をベースとした治療 - Google Patents

Ｃｒｉｓｐｒ／ｃａｓ９をベースとした治療 Download PDF

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JP2018520149A
JP2018520149A JP2017567629A JP2017567629A JP2018520149A JP 2018520149 A JP2018520149 A JP 2018520149A JP 2017567629 A JP2017567629 A JP 2017567629A JP 2017567629 A JP2017567629 A JP 2017567629A JP 2018520149 A JP2018520149 A JP 2018520149A
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dystrophy
corneal dystrophy
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nuclease
syndrome
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アルバート エス． ジュン，
アルバート エス． ジュン，
ビノッド ジャスクラ−ランガ，
ビノッド ジャスクラ−ランガ，
ドナルド ザック，
ドナルド ザック，
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ザ・ジョンズ・ホプキンス・ユニバーシティー
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    • A—HUMAN NECESSITIES
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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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
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JP2017567629A 2015-07-02 2016-07-05 Ｃｒｉｓｐｒ／ｃａｓ９をベースとした治療 Pending JP2018520149A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562188013P 2015-07-02 2015-07-02
US62/188,013 2015-07-02
PCT/US2016/040962 WO2017004616A1 (en) 2015-07-02 2016-07-05 Crispr/cas9-based treatments

Publications (2)

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JP2018520149A true JP2018520149A (ja) 2018-07-26
JP2018520149A5 JP2018520149A5 (https=) 2019-08-15

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US (1) US20200010854A1 (https=)
EP (1) EP3317409A4 (https=)
JP (1) JP2018520149A (https=)
KR (1) KR20180041120A (https=)
CN (1) CN108350446A (https=)
AU (1) AU2016287836A1 (https=)
BR (1) BR112017028201A2 (https=)
CA (1) CA2989331A1 (https=)
CL (1) CL2017003411A1 (https=)
EA (1) EA201890203A1 (https=)
IL (1) IL256279A (https=)
MX (1) MX2017016921A (https=)
WO (1) WO2017004616A1 (https=)

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US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
DK3155101T3 (da) 2014-06-16 2020-05-04 Univ Johns Hopkins Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
EP3445375A1 (en) * 2016-04-22 2019-02-27 Intellia Therapeutics, Inc. Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2020046861A1 (en) * 2018-08-27 2020-03-05 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
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WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CN111065736A (zh) * 2017-06-07 2020-04-24 国立大学法人东京大学 针对颗粒状角膜变性症的基因治疗药物
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
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KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
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JP2018534114A (ja) * 2015-11-13 2018-11-22 タラ ムーア 角膜ジストロフィーの治療方法
JP2021183626A (ja) * 2015-11-13 2021-12-02 タラ ムーア 角膜ジストロフィーの治療方法
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EA201890203A1 (ru) 2018-07-31
MX2017016921A (es) 2018-04-10
EP3317409A1 (en) 2018-05-09
CA2989331A1 (en) 2017-01-05
KR20180041120A (ko) 2018-04-23
CN108350446A (zh) 2018-07-31
AU2016287836A1 (en) 2018-02-15
CL2017003411A1 (es) 2018-08-17
BR112017028201A2 (pt) 2018-08-28
IL256279A (en) 2018-02-28
US20200010854A1 (en) 2020-01-09
EP3317409A4 (en) 2019-02-20
WO2017004616A1 (en) 2017-01-05

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