JP2016521975A - 遺伝的状態の処置のための方法および組成物 - Google Patents

遺伝的状態の処置のための方法および組成物 Download PDF

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Publication number
JP2016521975A
JP2016521975A JP2016514094A JP2016514094A JP2016521975A JP 2016521975 A JP2016521975 A JP 2016521975A JP 2016514094 A JP2016514094 A JP 2016514094A JP 2016514094 A JP2016514094 A JP 2016514094A JP 2016521975 A JP2016521975 A JP 2016521975A
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gene
cell
sequence
plant
domain
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JP2016521975A5 (enExample
Inventor
アントニー コンウェイ，
アントニー コンウェイ，
グレゴリー ジェイ． コスト，
グレゴリー ジェイ． コスト，
ラッセル デケルヴェル，
ラッセル デケルヴェル，
エドワード ジェイ． レバー，
エドワード ジェイ． レバー，
アンドレアス レイク，
アンドレアス レイク，
フョードル ウルノフ，
フョードル ウルノフ，
ジアンビン ワン，
ジアンビン ワン，
エイチ． スティーブ ザン，
エイチ． スティーブ ザン，
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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Publication of JP2016521975A publication Critical patent/JP2016521975A/ja
Publication of JP2016521975A5 publication Critical patent/JP2016521975A5/ja
Priority to JP2019010055A priority Critical patent/JP6964103B2/ja
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2/00—Peptides of undefined number of amino acids; Derivatives thereof
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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
    • C—CHEMISTRY; METALLURGY
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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
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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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/30—Special therapeutic applications
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)

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  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
JP2016514094A 2013-05-15 2014-05-15 遺伝的状態の処置のための方法および組成物 Withdrawn JP2016521975A (ja)

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US201361823689P 2013-05-15 2013-05-15
US61/823,689 2013-05-15
PCT/US2014/038216 WO2014186585A2 (en) 2013-05-15 2014-05-15 Methods and compositions for treatment of a genetic condition

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JP2020087329A Division JP2020120690A (ja) 2013-05-15 2020-05-19 遺伝的状態の処置のための方法および組成物

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JP2016514094A Withdrawn JP2016521975A (ja) 2013-05-15 2014-05-15 遺伝的状態の処置のための方法および組成物
JP2019010055A Active JP6964103B2 (ja) 2013-05-15 2019-01-24 遺伝的状態の処置のための方法および組成物
JP2020087329A Withdrawn JP2020120690A (ja) 2013-05-15 2020-05-19 遺伝的状態の処置のための方法および組成物
JP2022090175A Ceased JP2022107776A (ja) 2013-05-15 2022-06-02 遺伝的状態の処置のための方法および組成物

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US (4) US9873894B2 (enExample)
EP (2) EP3730615A3 (enExample)
JP (4) JP2016521975A (enExample)
CN (2) CN105683376A (enExample)
AU (1) AU2014265331B2 (enExample)
CA (1) CA2910489A1 (enExample)
HK (1) HK1223401A1 (enExample)
WO (1) WO2014186585A2 (enExample)

Cited By (40)

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JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
WO2019098759A3 (ko) * 2017-11-16 2019-07-18 재단법인 목암생명과학연구소 형질전환된 인간세포 및 이의 용도
US10494621B2 (en) 2015-06-18 2019-12-03 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
US10550372B2 (en) 2013-12-12 2020-02-04 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US10577630B2 (en) 2013-06-17 2020-03-03 The Broad Institute, Inc. Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
US10696986B2 (en) 2014-12-12 2020-06-30 The Board Institute, Inc. Protected guide RNAS (PGRNAS)
US10711285B2 (en) 2013-06-17 2020-07-14 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US10711256B2 (en) 2012-04-27 2020-07-14 Duke University Genetic correction of mutated genes
WO2020149395A1 (ja) * 2019-01-18 2020-07-23 国立大学法人大阪大学 栄養障害型表皮水疱症治療薬
US10781444B2 (en) 2013-06-17 2020-09-22 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US10851357B2 (en) 2013-12-12 2020-12-01 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
JP2021500072A (ja) * 2017-10-17 2021-01-07 クリスパー セラピューティクス アクチェンゲゼルシャフト 血友病ａに対する遺伝子編集用組成物及び方法
US10930367B2 (en) 2012-12-12 2021-02-23 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for Cas enzymes or CRISPR-Cas systems for target sequences and conveying results thereof
US10946108B2 (en) 2013-06-17 2021-03-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US11155795B2 (en) 2013-12-12 2021-10-26 The Broad Institute, Inc. CRISPR-Cas systems, crystal structure and uses thereof
JP2022505173A (ja) * 2018-10-17 2022-01-14 クリスパー・セラピューティクス・アクチェンゲゼルシャフト 導入遺伝子を送達するための組成物および方法
JP2022512731A (ja) * 2018-10-18 2022-02-07 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
JP2022514956A (ja) * 2018-12-23 2022-02-16 シーエスエル・ベーリング・エルエルシー 細胞調節剤の送達のためのナノカプセル
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US11427817B2 (en) 2015-08-25 2022-08-30 Duke University Compositions and methods of improving specificity in genomic engineering using RNA-guided endonucleases
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JP2023520262A (ja) * 2019-06-05 2023-05-17 クリスパー セラピューティクス アクチェンゲゼルシャフト 改善された第ｖｉｉｉ因子発現による血友病ａのための遺伝子編集
JP2023531729A (ja) * 2020-06-26 2023-07-25 シーエスエル・ベーリング・エルエルシー キルスイッチを有するドナーｔ細胞
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