JP2018513681A - 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系 - Google Patents

細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系 Download PDF

Info

Publication number
JP2018513681A
JP2018513681A JP2017552079A JP2017552079A JP2018513681A JP 2018513681 A JP2018513681 A JP 2018513681A JP 2017552079 A JP2017552079 A JP 2017552079A JP 2017552079 A JP2017552079 A JP 2017552079A JP 2018513681 A JP2018513681 A JP 2018513681A
Authority
JP
Japan
Prior art keywords
protein
sequence
dna
integrase
cas9
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.)
Pending
Application number
JP2017552079A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018513681A5 (enExample
Inventor
シャイフ,フェルフ
川村哲也
モー,グロリア
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.)
Exeligen Scientific inc
Original Assignee
Exeligen Scientific 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 Exeligen Scientific inc filed Critical Exeligen Scientific inc
Publication of JP2018513681A publication Critical patent/JP2018513681A/ja
Publication of JP2018513681A5 publication Critical patent/JP2018513681A5/ja
Priority to JP2021110120A priority Critical patent/JP2021176301A/ja
Priority to JP2023121860A priority patent/JP7599740B2/ja
Priority to JP2024205951A priority patent/JP2025065094A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226Class 2 CAS enzyme complex, e.g. single CAS protein
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2017552079A 2015-03-31 2016-03-31 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系 Pending JP2018513681A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021110120A JP2021176301A (ja) 2015-03-31 2021-07-01 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2023121860A JP7599740B2 (ja) 2015-03-31 2023-07-26 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2024205951A JP2025065094A (ja) 2015-03-31 2024-11-27 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562140454P 2015-03-31 2015-03-31
US62/140,454 2015-03-31
US201562210451P 2015-08-27 2015-08-27
US62/210,451 2015-08-27
US201562240359P 2015-10-12 2015-10-12
US62/240,359 2015-10-12
PCT/US2016/025426 WO2016161207A1 (en) 2015-03-31 2016-03-31 Cas 9 retroviral integrase and cas 9 recombinase systems for targeted incorporation of a dna sequence into a genome of a cell or organism

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021110120A Division JP2021176301A (ja) 2015-03-31 2021-07-01 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系

Publications (2)

Publication Number Publication Date
JP2018513681A true JP2018513681A (ja) 2018-05-31
JP2018513681A5 JP2018513681A5 (enExample) 2019-05-09

Family

ID=55745849

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2017552079A Pending JP2018513681A (ja) 2015-03-31 2016-03-31 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2021110120A Pending JP2021176301A (ja) 2015-03-31 2021-07-01 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2023121860A Active JP7599740B2 (ja) 2015-03-31 2023-07-26 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2024205951A Pending JP2025065094A (ja) 2015-03-31 2024-11-27 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2021110120A Pending JP2021176301A (ja) 2015-03-31 2021-07-01 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2023121860A Active JP7599740B2 (ja) 2015-03-31 2023-07-26 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
JP2024205951A Pending JP2025065094A (ja) 2015-03-31 2024-11-27 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系

Country Status (8)

Country Link
US (2) US20180080051A1 (enExample)
EP (2) EP4600366A3 (enExample)
JP (4) JP2018513681A (enExample)
KR (1) KR102769515B1 (enExample)
CN (1) CN108124453B (enExample)
DK (1) DK3277805T3 (enExample)
ES (1) ES3030433T3 (enExample)
WO (1) WO2016161207A1 (enExample)

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022513376A (ja) * 2018-10-22 2022-02-07 ユニバーシティ オブ ロチェスター レトロウイルスインテグラーゼ-Cas9融合タンパク質を使用した指向性非相同DNA挿入によるゲノム編集
JP2022521432A (ja) * 2019-02-25 2022-04-07 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランスアクチベーター及びその使用
JP2022532939A (ja) * 2019-05-23 2022-07-20 クリスティアナ ケア ヘルス サービシズ,インコーポレーテッド がんを処置するためのnrf2の遺伝子ノックアウト
JP2022533268A (ja) * 2019-05-23 2022-07-21 クリスティアナ ケア ヘルス サービシズ,インコーポレーテッド がんを処置するためのバリアントnrf2の遺伝子ノックアウト
JP2022540318A (ja) * 2019-06-11 2022-09-15 ウニベルシタット ポンペウ ファブラ 標的遺伝子編集構築物およびそれを使用する方法
JP2023535025A (ja) * 2020-07-24 2023-08-15 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランス活性化因子およびその使用
JP2023553701A (ja) * 2020-12-16 2023-12-25 ウニベルシタット ポンペウ ファブラ 先天性筋ジストロフィーの処置のための治療用lama2ペイロード
JP2023554504A (ja) * 2020-12-16 2023-12-27 ユニバーシタット ポンペウ ファブラ プログラム可能なトランスポザーゼおよびその使用

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
EP4600366A3 (en) * 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3433363A1 (en) 2016-03-25 2019-01-30 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
GB201610041D0 (en) * 2016-06-08 2016-07-20 Oxford Genetics Ltd Methods
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US12188059B2 (en) 2016-09-13 2025-01-07 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
CA3039409A1 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
JP2019535287A (ja) * 2016-11-22 2019-12-12 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド Crispr/cpf1システム及び方法
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
KR102523318B1 (ko) 2016-12-16 2023-04-18 비-모젠 바이오테크놀로지스, 인크. 증진된 hAT 패밀리 트랜스포존 매개 유전자 전달 및 연관된 조성물, 시스템, 및 방법
US11278570B2 (en) 2016-12-16 2022-03-22 B-Mogen Biotechnologies, Inc. Enhanced hAT family transposon-mediated gene transfer and associated compositions, systems, and methods
US12286727B2 (en) 2016-12-19 2025-04-29 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
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
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
EP3650540B1 (en) 2017-07-07 2025-04-16 Toolgen Incorporated Target-specific crispr mutant
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
WO2019090174A1 (en) * 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3810764A2 (en) 2018-06-21 2021-04-28 B-Mogen Biotechnologies, Inc. Enhanced hat family transposon-mediated gene transfer and associated compositions, systems, and methods
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020131862A1 (en) * 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020181264A1 (en) * 2019-03-07 2020-09-10 The Trustees Of Columbia University In The City Of New York Rna-guided dna integration using tn7-like transposons
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020243085A1 (en) * 2019-05-24 2020-12-03 The Trustees Of Columbia University In The City Of New York Engineered cas-transposon system for programmable and site-directed dna transpositions
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20220403357A1 (en) * 2019-11-12 2022-12-22 The Broad Institute, Inc. Small type ii cas proteins and methods of use thereof
US20230257771A1 (en) * 2020-04-20 2023-08-17 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
KR102679172B1 (ko) * 2020-07-06 2024-06-28 한국과학기술연구원 질환 세포-특이적인 miRNA에 의해 세포 생리 활성 조절 물질의 활성을 조절하는 복합체 및 이를 CRISPR/Cas 시스템에 적용한 질환 특이적 유전자 조작용 복합체
WO2022007959A1 (zh) * 2020-07-10 2022-01-13 中国科学院动物研究所 一种编辑核酸的系统及方法
CN112159822A (zh) * 2020-09-30 2021-01-01 扬州大学 一种PS转座酶与CRISPR/dCpf1融合蛋白表达载体及其介导的定点整合方法
KR102803305B1 (ko) * 2020-11-25 2025-05-07 연세대학교 산학협력단 트랜스-스플라이싱 아데노-연관 바이러스 벡터를 포함하는 프라임에디팅용 조성물
WO2024227131A1 (en) * 2023-04-27 2024-10-31 Rensselaer Polytechnic Institute Recombinant enzyme for the accurate insertion of dna sequences in eukaryotic cells
CN118497204B (zh) * 2024-07-16 2024-12-03 西北农林科技大学深圳研究院 一种质粒CRISPR-pCas9n及基因编辑的方法与应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060252140A1 (en) * 2005-04-29 2006-11-09 Yant Stephen R Development of a transposon system for site-specific DNA integration in mammalian cells
US20140193915A1 (en) * 2012-12-18 2014-07-10 Monsanto Technology, Llc Compositions and methods for custom site-specific dna recombinases
WO2015035162A2 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9 variants and uses thereof

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0871711A1 (en) * 1995-12-01 1998-10-21 The Regents Of The University Of California Compositions and methods for site-directed integration into dna
US6080849A (en) 1997-09-10 2000-06-27 Vion Pharmaceuticals, Inc. Genetically modified tumor-targeted bacteria with reduced virulence
WO1999045132A1 (en) 1998-03-02 1999-09-10 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US20040003420A1 (en) 2000-11-10 2004-01-01 Ralf Kuhn Modified recombinase
GB0400814D0 (en) 2004-01-14 2004-02-18 Ark Therapeutics Ltd Integrating gene therapy vector
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
EP2092068B1 (en) 2006-12-14 2014-10-08 Dow AgroSciences LLC Optimized non-canonical zinc finger proteins
US8816153B2 (en) 2010-08-27 2014-08-26 Monsanto Technology Llc Recombinant DNA constructs employing site-specific recombination
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
ES2750550T3 (es) 2013-03-01 2020-03-26 Univ Minnesota Corrección de gen a base de TALEN
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
DK3004337T3 (da) * 2013-05-29 2017-11-13 Cellectis Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system
US11685935B2 (en) * 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
CN104404036B (zh) * 2014-11-03 2017-12-01 赛业(苏州)生物科技有限公司 基于CRISPR/Cas9技术的条件性基因敲除方法
EP4600366A3 (en) * 2015-03-31 2025-10-22 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060252140A1 (en) * 2005-04-29 2006-11-09 Yant Stephen R Development of a transposon system for site-specific DNA integration in mammalian cells
US20140193915A1 (en) * 2012-12-18 2014-07-10 Monsanto Technology, Llc Compositions and methods for custom site-specific dna recombinases
WO2015035162A2 (en) * 2013-09-06 2015-03-12 President And Fellows Of Harvard College Cas9 variants and uses thereof

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
J. VIROL., 1997, VOL. 71, NO. 1, PP. 458-464, JPN6020009965, ISSN: 0004669329 *
METHODS, 2009, VOL. 47, PP. 269-276, JPN6020009966, ISSN: 0004669330 *

Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022513376A (ja) * 2018-10-22 2022-02-07 ユニバーシティ オブ ロチェスター レトロウイルスインテグラーゼ-Cas9融合タンパク質を使用した指向性非相同DNA挿入によるゲノム編集
JP7558575B2 (ja) 2018-10-22 2024-10-01 ユニバーシティ オブ ロチェスター レトロウイルスインテグラーゼ-Cas9融合タンパク質を使用した指向性非相同DNA挿入によるゲノム編集
JP2022521432A (ja) * 2019-02-25 2022-04-07 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランスアクチベーター及びその使用
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランスアクチベーター及びその使用
JP2022532939A (ja) * 2019-05-23 2022-07-20 クリスティアナ ケア ヘルス サービシズ,インコーポレーテッド がんを処置するためのnrf2の遺伝子ノックアウト
JP2022533268A (ja) * 2019-05-23 2022-07-21 クリスティアナ ケア ヘルス サービシズ,インコーポレーテッド がんを処置するためのバリアントnrf2の遺伝子ノックアウト
JP7585237B2 (ja) 2019-05-23 2024-11-18 クリスティアナ ケア ジーン エディティング インスティテュート,インコーポレーテッド がんを処置するためのバリアントnrf2の遺伝子ノックアウト
JP2022540318A (ja) * 2019-06-11 2022-09-15 ウニベルシタット ポンペウ ファブラ 標的遺伝子編集構築物およびそれを使用する方法
JP2023535025A (ja) * 2020-07-24 2023-08-15 ユニバーシティ オブ マサチューセッツ Dna結合ドメイントランス活性化因子およびその使用
JP2023553701A (ja) * 2020-12-16 2023-12-25 ウニベルシタット ポンペウ ファブラ 先天性筋ジストロフィーの処置のための治療用lama2ペイロード
JP2023554504A (ja) * 2020-12-16 2023-12-27 ユニバーシタット ポンペウ ファブラ プログラム可能なトランスポザーゼおよびその使用

Also Published As

Publication number Publication date
KR102769515B1 (ko) 2025-02-18
WO2016161207A1 (en) 2016-10-06
JP7599740B2 (ja) 2024-12-16
EP4600366A2 (en) 2025-08-13
US20220315952A1 (en) 2022-10-06
KR20250002825A (ko) 2025-01-07
CN108124453B (zh) 2022-04-05
DK3277805T3 (da) 2025-06-10
US20180080051A1 (en) 2018-03-22
JP2021176301A (ja) 2021-11-11
EP3277805A1 (en) 2018-02-07
CN108124453A (zh) 2018-06-05
JP2023156355A (ja) 2023-10-24
JP2025065094A (ja) 2025-04-17
EP4600366A3 (en) 2025-10-22
EP3277805B1 (en) 2025-05-07
ES3030433T3 (en) 2025-06-30
KR20180029953A (ko) 2018-03-21

Similar Documents

Publication Publication Date Title
JP7599740B2 (ja) 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系
US11555181B2 (en) Engineered cascade components and cascade complexes
JP7700079B2 (ja) ゲノム編集のためのシステム及び方法
US20210403861A1 (en) Nucleotide-specific recognition sequences for designer tal effectors
KR20190005801A (ko) 표적 특이적 crispr 변이체
AU2019222568A1 (en) Engineered Cas9 systems for eukaryotic genome modification
WO2017107898A2 (en) Compositions and methods for gene editing
CN110997909A (zh) 改进的转座酶多肽及其用途
WO2019041344A1 (en) METHODS AND COMPOSITIONS FOR THE TRANSFECTION OF SINGLE STRANDED DNA
CN113874510A (zh) 包括具有β滑移突变的人源化TTR基因座的非人动物和使用方法
US20210108188A1 (en) Non-covalent systems and methods for dna editing
KR102894634B1 (ko) 세포 또는 유기체의 게놈으로의 DNA 서열의 표적화 혼입을 위한 Cas 9 레트로바이러스 인테그라제 시스템 및 Cas 9 재조합효소 시스템
US20250145973A1 (en) Conditioned dna modifying enzyme comprising heterologous dna binding domain
WO2025128981A1 (en) Compositions and methods for genome editing
KR20250171479A (ko) 세포 또는 유기체의 게놈으로의 DNA 서열의 표적화 혼입을 위한 Cas 9 레트로바이러스 인테그라제 시스템 및 Cas 9 재조합효소 시스템
Qi et al. Construction of a TAT-Cas9-EGFP Site-Specific Integration Eukaryotic Cell Line Using Efficient PEG10 Modification
KR20230136479A (ko) 동물세포의 유전자 교정을 위한 CRISPR-Cas9 벡터 개발
WO2024235991A1 (en) Rna-guided nucleases and nucleic acid targeting systems comprising such rna-guided nucleases
WO2024042168A1 (en) Novel rna-guided nucleases and nucleic acid targeting systems comprising such rna-guided nucleases
CN115896037A (zh) 一种稳定敲除mettl7b基因的脑胶质瘤重组细胞及其构建方法
BR122025004697A2 (pt) Sistema para modificar uma sequência cromossômica em uma célula eucariótica, pluralidade de ácidos nucleicos, vetor, célula eucariótica, proteína de fusão, composição e usos
HK1254126B (zh) 三组分crispr/cas复合系统及其用途

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190328

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190328

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200317

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200617

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200817

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200917

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210302

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210701

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20210701

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20210713

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20210906

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20210907

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20211007

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20211007

RD02 Notification of acceptance of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7422

Effective date: 20211129

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20211129

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211125

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20211224

C211 Notice of termination of reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C211

Effective date: 20211228

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20220511

C13 Notice of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: C13

Effective date: 20230215

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20230405