JP7033583B2 - ゲノム編集効率を高めるための方法、組成物及びキット - Google Patents

ゲノム編集効率を高めるための方法、組成物及びキット Download PDF

Info

Publication number
JP7033583B2
JP7033583B2 JP2019501575A JP2019501575A JP7033583B2 JP 7033583 B2 JP7033583 B2 JP 7033583B2 JP 2019501575 A JP2019501575 A JP 2019501575A JP 2019501575 A JP2019501575 A JP 2019501575A JP 7033583 B2 JP7033583 B2 JP 7033583B2
Authority
JP
Japan
Prior art keywords
alkyl
atoms
compound
cells
cycloalkyl
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2019501575A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019521139A (ja
JP2019521139A5 (cg-RX-API-DMAC7.html
Inventor
ノーゼハン アブドゥル－マナン，
デイビッド エー． ニューサム，
ジャック ズワレン，
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Vertex Pharmaceuticals Inc
Original Assignee
Vertex Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Vertex Pharmaceuticals Inc filed Critical Vertex Pharmaceuticals Inc
Publication of JP2019521139A publication Critical patent/JP2019521139A/ja
Publication of JP2019521139A5 publication Critical patent/JP2019521139A5/ja
Application granted granted Critical
Publication of JP7033583B2 publication Critical patent/JP7033583B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • 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
    • 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]
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Adhesives Or Adhesive Processes (AREA)
  • Document Processing Apparatus (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2019501575A 2016-07-13 2017-07-13 ゲノム編集効率を高めるための方法、組成物及びキット Active JP7033583B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662361961P 2016-07-13 2016-07-13
US201662361781P 2016-07-13 2016-07-13
US62/361,781 2016-07-13
US62/361,961 2016-07-13
PCT/US2017/041979 WO2018013840A1 (en) 2016-07-13 2017-07-13 Methods, compositions and kits for increasing genome editing efficiency

Publications (3)

Publication Number Publication Date
JP2019521139A JP2019521139A (ja) 2019-07-25
JP2019521139A5 JP2019521139A5 (cg-RX-API-DMAC7.html) 2020-08-20
JP7033583B2 true JP7033583B2 (ja) 2022-03-10

Family

ID=59388213

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019501575A Active JP7033583B2 (ja) 2016-07-13 2017-07-13 ゲノム編集効率を高めるための方法、組成物及びキット

Country Status (9)

Country Link
US (2) US11124805B2 (cg-RX-API-DMAC7.html)
EP (2) EP3484870B1 (cg-RX-API-DMAC7.html)
JP (1) JP7033583B2 (cg-RX-API-DMAC7.html)
CN (1) CN109863143B (cg-RX-API-DMAC7.html)
AU (2) AU2017295720B2 (cg-RX-API-DMAC7.html)
CA (1) CA3030783A1 (cg-RX-API-DMAC7.html)
ES (1) ES2938210T3 (cg-RX-API-DMAC7.html)
MA (1) MA45670A (cg-RX-API-DMAC7.html)
WO (1) WO2018013840A1 (cg-RX-API-DMAC7.html)

Families Citing this family (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
EP3092049A1 (en) 2014-01-08 2016-11-16 Flodesign Sonics Inc. Acoustophoresis device with dual acoustophoretic chamber
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US11667911B2 (en) 2015-09-24 2023-06-06 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/CAS-mediated genome editing
CR20180323A (es) 2015-11-20 2018-08-06 Idorsia Pharmaceuticals Ltd Derivados de indol n-sustituídos como moduladores de los receptores de pge2
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
CN109863143B (zh) 2016-07-13 2021-10-15 威泰克斯制药公司 提高基因组编辑效率的方法、组合物和试剂盒
KR20190127655A (ko) 2016-10-19 2019-11-13 프로디자인 소닉스, 인크. 음향학에 의한 친화성 세포 추출
CA3063632A1 (en) 2017-05-18 2018-11-22 Idorsia Pharmaceuticals Ltd Benzofurane and benzothiophene derivatives as pge2 receptor modulators
ES2896476T3 (es) 2017-05-18 2022-02-24 Idorsia Pharmaceuticals Ltd Derivados de pirimidina
WO2018210994A1 (en) 2017-05-18 2018-11-22 Idorsia Pharmaceuticals Ltd Phenyl derivatives as pge2 receptor modulators
KR102626982B1 (ko) 2017-05-18 2024-01-18 이도르시아 파마슈티컬스 리미티드 Pge2 수용체 조절제로서의 피리미딘 유도체
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP2021507561A (ja) 2017-12-14 2021-02-22 フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． 音響トランスデューサドライバ及びコントローラ
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
EP3740483B1 (en) 2018-01-17 2023-01-11 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
JP2021522819A (ja) * 2018-05-07 2021-09-02 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 インビトロベータ細胞分化に関連する細胞集団および遺伝子発現
WO2019224603A1 (en) 2018-05-24 2019-11-28 Crispr Therapeutics Ag Methods and compositions for efficient gene deletion
CN110592141B (zh) * 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
EP3640329A1 (en) * 2018-10-18 2020-04-22 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Gene editing based cancer treatment
WO2020120711A1 (en) 2018-12-12 2020-06-18 Depixus Method of nucleic acid enrichment using site-specific nucleases followed by capture
WO2020205664A1 (en) * 2019-03-29 2020-10-08 Youhealth Biotech, Limited Compositions and methods for cellular reprogramming to rescue visual function
WO2020227255A1 (en) * 2019-05-06 2020-11-12 The Regents Of The University Of Michigan Targeted therapy
AU2020337919A1 (en) 2019-08-27 2022-03-24 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive DNA
CN112553330B (zh) * 2019-09-26 2022-07-26 首都医科大学附属北京友谊医院 新型肿瘤相关转录因子zscan16及其在抑制肿瘤中的应用
US20220340936A1 (en) * 2019-09-27 2022-10-27 The Broad Institute, Inc. Programmable polynucleotide editors for enhanced homologous recombination
EP4051787A1 (en) 2019-11-01 2022-09-07 Sangamo Therapeutics, Inc. Gin recombinase variants
CN110863011A (zh) * 2019-11-28 2020-03-06 浙江大学 干扰猪crtc3表达的质粒及腺病毒的构建方法及其用途
CN112877364B (zh) * 2019-11-29 2023-07-28 中国医学科学院药物研究所 软骨下骨细胞向关节软骨细胞直接转化的重编程诱导方案
CN111088290B (zh) * 2019-12-30 2021-10-08 同济大学 一种杜鹃素在基因编辑中的应用
WO2021166306A1 (en) * 2020-02-19 2021-08-26 Kyoto University Production method of genome-edited cells and kit therefor
EP4153741A1 (en) * 2020-05-21 2023-03-29 Oxford Genetics Limited Hdr enhancers
GB202008201D0 (en) * 2020-06-01 2020-07-15 Neophore Ltd Inhibitor compounds
CN113897357A (zh) * 2020-07-06 2022-01-07 北京大学 Twist1基因编辑系统及其在制备治疗三阴性乳腺癌的产品中的应用
CN114717232A (zh) * 2020-12-22 2022-07-08 未来智人再生医学研究院(广州)有限公司 表达irf-1靶向抑制因子的多能干细胞及其衍生物与应用
CN112750498B (zh) * 2020-12-30 2022-06-24 同济大学 靶向逆转录引物结合位点从而抑制hiv病毒复制的方法
TW202302848A (zh) 2021-02-26 2023-01-16 美商維泰克斯製藥公司 以crispr/sacas9治療第1型肌強直性營養不良之組合物及方法
TW202246510A (zh) 2021-02-26 2022-12-01 美商維泰克斯製藥公司 以crispr/slucas9治療第1型肌強直性營養不良之組合物及方法
CN113373148B (zh) * 2021-06-16 2022-08-26 中国人民解放军军事科学院军事医学研究院 一种调控app表达的靶标位点序列及其在防治ad上的应用
WO2023018637A1 (en) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements
EP4423278A4 (en) * 2021-10-29 2025-10-22 Univ Duke COMPOSITIONS AND SYSTEMS FOR PROGRAMMABLE RNA-BASED CELL EDITING AND METHODS OF MAKING AND USING THE SAME
CN114277119B (zh) * 2021-12-09 2023-05-16 浙江大学医学院附属邵逸夫医院 一种环状RNA circ-Arsb在制备防治骨质疏松产品中的应用
WO2023120530A1 (ja) * 2021-12-24 2023-06-29 国立大学法人大阪大学 相同組換えを利用したゲノム編集細胞の製造方法
US20250115872A1 (en) * 2022-02-01 2025-04-10 The Brigham And Women's Hospital, Inc. Generation of brown adipocytes from human pluripotent stem cells
CN114524811B (zh) * 2022-03-16 2025-01-24 中国人民解放军军事科学院军事医学研究院 Lxh0307、lxh0308作为基因编辑的小分子抑制剂及其应用
CN114685494B (zh) * 2022-03-16 2024-04-16 中国人民解放军军事科学院军事医学研究院 SpCas9抑制剂的合成制备方法及其应用
CN116004542A (zh) * 2022-09-26 2023-04-25 苏州吉纳星辰生物技术有限公司 一种提高基因敲入效率的方法及其应用
WO2024238691A2 (en) * 2023-05-15 2024-11-21 The Regents Of The University Of California Andp gene editing system
US20250099613A1 (en) * 2023-07-26 2025-03-27 University Of Utah Research Foundation Compositions And Methods For Regulating ZNF865
CN119215053B (zh) * 2024-12-05 2025-04-01 南昌大学 Ye6144在制备治疗乳腺癌引起的骨骼肌萎缩药物中的应用
CN119752995B (zh) * 2024-12-26 2025-11-28 中国农业科学院棉花研究所 GhNFXL1基因在提高棉属植物耐盐性和/或耐冷性中的应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015058067A1 (en) 2013-10-17 2015-04-23 Vertex Pharmaceuticals Incorporated Co-crystals of (s)-n-methyl-8-(1-((2'-methyl-[4,5'-bipyrimidin]-6-yl)amino)propan-2-yl)quinoline-4-carboxamide and deuterated derivatives thereof as dna-pk inhibitors
JP2015514804A (ja) 2012-04-24 2015-05-21 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｄｎａ−ｐｋ阻害剤
JP2015534817A (ja) 2012-11-01 2015-12-07 ファクター バイオサイエンス インコーポレイテッド 細胞中でタンパク質を発現するための方法および生成物
WO2016081923A2 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Family Cites Families (92)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
US3773919A (en) 1969-10-23 1973-11-20 Du Pont Polylactide-drug mixtures
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
US6479626B1 (en) 1998-03-02 2002-11-12 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
EP1236045B1 (en) 1999-12-06 2005-11-09 Sangamo Biosciences Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
CA2398590C (en) 2000-02-08 2012-08-28 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
JP4968498B2 (ja) 2002-01-23 2012-07-04 ユニバーシティ オブ ユタ リサーチ ファウンデーション ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2562193A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
ATE462783T1 (de) 2006-05-25 2010-04-15 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
WO2008157688A2 (en) 2007-06-19 2008-12-24 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap
WO2010017562A2 (en) 2008-08-08 2010-02-11 Mayo Foundation For Medical Education And Research Induced pluripotent stem cells
EP2615106B1 (en) 2010-02-08 2018-04-25 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
JP6490426B2 (ja) 2012-02-29 2019-03-27 サンガモ セラピューティクス， インコーポレイテッド ハンチントン病を治療するための方法および組成物
EP3808844A1 (en) 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
US9181535B2 (en) 2012-09-24 2015-11-10 The Chinese University Of Hong Kong Transcription activator-like effector nucleases (TALENs)
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
KR20150105635A (ko) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
WO2014093709A1 (en) 2012-12-12 2014-06-19 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
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
PL2771468T3 (pl) 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US20140186919A1 (en) 2012-12-12 2014-07-03 Feng Zhang Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
KR102874079B1 (ko) * 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
EP2986709A4 (en) 2013-04-16 2017-03-15 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN106062197A (zh) 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
EP3620524A1 (en) 2013-06-17 2020-03-11 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
KR101448678B1 (ko) 2013-08-12 2014-10-08 김형기 단추 재봉실의 뿌리감기 장치
US20150095137A1 (en) 2013-09-27 2015-04-02 Stout Partners LP System and apparatus for effective coordination and scheduling of accesses to rate limited online sites to obtain data for use in assessing social metrics based on domain tailored evaluation of social media exposure
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
CN109863143B (zh) 2016-07-13 2021-10-15 威泰克斯制药公司 提高基因组编辑效率的方法、组合物和试剂盒

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015514804A (ja) 2012-04-24 2015-05-21 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｄｎａ−ｐｋ阻害剤
JP2015534817A (ja) 2012-11-01 2015-12-07 ファクター バイオサイエンス インコーポレイテッド 細胞中でタンパク質を発現するための方法および生成物
WO2015058067A1 (en) 2013-10-17 2015-04-23 Vertex Pharmaceuticals Incorporated Co-crystals of (s)-n-methyl-8-(1-((2'-methyl-[4,5'-bipyrimidin]-6-yl)amino)propan-2-yl)quinoline-4-carboxamide and deuterated derivatives thereof as dna-pk inhibitors
WO2016081923A2 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Genome Medicine，2015年，7: 93，pp. 1-11

Also Published As

Publication number Publication date
WO2018013840A1 (en) 2018-01-18
US20220106615A1 (en) 2022-04-07
AU2021232813A1 (en) 2021-10-14
ES2938210T3 (es) 2023-04-05
AU2017295720B2 (en) 2021-07-22
US20190225990A1 (en) 2019-07-25
US11124805B2 (en) 2021-09-21
US12031150B2 (en) 2024-07-09
EP3484870A1 (en) 2019-05-22
JP2019521139A (ja) 2019-07-25
CN109863143A (zh) 2019-06-07
CA3030783A1 (en) 2018-01-18
AU2017295720A1 (en) 2019-02-07
EP3484870B1 (en) 2022-11-16
MA45670A (fr) 2019-05-22
EP4219462A1 (en) 2023-08-02
AU2021232813B2 (en) 2023-04-13
CN109863143B (zh) 2021-10-15

Similar Documents

Publication Publication Date Title
JP7033583B2 (ja) ゲノム編集効率を高めるための方法、組成物及びキット
JP7609952B2 (ja) Ｄｎａ－ｐｋ阻害剤
JP7483612B2 (ja) ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット
US12005127B2 (en) DNA-PK inhibitors
HK40096539A (en) Methods, compositions and kits for increasing genome editing efficiency
EA042641B1 (ru) Хиноксалиноновые соединения, композиции, способы и наборы для повышения эффективности редактирования генома
HK40039262B (en) Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
HK40039262A (en) Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
HK40002405B (en) Methods, compositions and kits for increasing genome editing efficiency
HK40002405A (en) Methods, compositions and kits for increasing genome editing efficiency
HK40039261B (en) Dna-pk inhibitors
HK40039261A (en) Dna-pk inhibitors

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200713

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200713

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210617

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210917

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220217

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220228

R150 Certificate of patent or registration of utility model

Ref document number: 7033583

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250