ES3030433T3 - Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell - Google Patents

Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell

Info

Publication number
ES3030433T3
ES3030433T3 ES16715977T ES16715977T ES3030433T3 ES 3030433 T3 ES3030433 T3 ES 3030433T3 ES 16715977 T ES16715977 T ES 16715977T ES 16715977 T ES16715977 T ES 16715977T ES 3030433 T3 ES3030433 T3 ES 3030433T3
Authority
ES
Spain
Prior art keywords
sequence
dna
protein
seq
integrase
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
ES16715977T
Other languages
English (en)
Spanish (es)
Inventor
Ferrukh Sheikh
Tetsuya Kawamura
Gloria Mo
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.)
Sohm Inc
Original Assignee
Sohm 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 Sohm Inc filed Critical Sohm Inc
Application granted granted Critical
Publication of ES3030433T3 publication Critical patent/ES3030433T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • C12N15/11DNA 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
    • 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
    • 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)
ES16715977T 2015-03-31 2016-03-31 Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell Active ES3030433T3 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562140454P 2015-03-31 2015-03-31
US201562210451P 2015-08-27 2015-08-27
US201562240359P 2015-10-12 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

Publications (1)

Publication Number Publication Date
ES3030433T3 true ES3030433T3 (en) 2025-06-30

Family

ID=55745849

Family Applications (1)

Application Number Title Priority Date Filing Date
ES16715977T Active ES3030433T3 (en) 2015-03-31 2016-03-31 Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell

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)

Families Citing this family (75)

* 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
MX2021004602A (es) * 2018-10-22 2021-09-08 Univ Rochester Edición del genoma por inserción no homóloga de adn dirigida utilizando una proteína de fusión retroviral integrasacas9.
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
SG11202108568YA (en) * 2019-02-25 2021-09-29 Univ Massachusetts Dna-binding domain transactivators 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
US12203070B2 (en) * 2019-05-23 2025-01-21 Christiana Care Gene Editing Institute, Inc. Gene knockout of NRF2 for treatment of cancer
US11505797B2 (en) 2019-05-23 2022-11-22 Christiana Care Health Services, Inc. Gene knockout of variant NRF2 for treatment of cancer
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
AU2020290790A1 (en) * 2019-06-11 2022-01-27 Universitat Pompeu Fabra Targeted gene editing constructs and methods of using the same
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
TW202221119A (zh) * 2020-07-24 2022-06-01 麻州大學 Dna結合域轉活化子及其用途
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
EP4263818A1 (en) * 2020-12-16 2023-10-25 Universitat Pompeu Fabra Therapeutic lama2 payload for treatment of congenital muscular dystrophy
KR20230123492A (ko) * 2020-12-16 2023-08-23 우니베르시타트 폼페우 파브라 프로그래밍 가능한 트랜스포사제 및 이의 용도
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

Family Cites Families (23)

* 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
US20060252140A1 (en) * 2005-04-29 2006-11-09 Yant Stephen R Development of a transposon system for site-specific DNA integration in mammalian cells
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
US9708589B2 (en) 2012-12-18 2017-07-18 Monsanto Technology Llc Compositions and methods for custom site-specific DNA recombinases
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
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
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

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
JP2018513681A (ja) 2018-05-31
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
KR20180029953A (ko) 2018-03-21

Similar Documents

Publication Publication Date Title
ES3030433T3 (en) Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
US11555181B2 (en) Engineered cascade components and cascade complexes
DK2462230T3 (en) METHODS AND COMPOSITIONS FOR TARGETED RE-MODIFICATION
US20220170013A1 (en) T:a to a:t base editing through adenosine methylation
EP2806025B1 (en) Use of zinc finger nucleases to stimulate gene targeting
CN110612353A (zh) 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
BR112020010479A2 (pt) sistemas cas9 geneticamente modificados para modificação de genoma eucariótico
US11332749B2 (en) Real-time reporter systems for monitoring base editing
EP3847251A1 (en) Compositions and methods for improved nucleases
KR20220016869A (ko) 베타-슬립 돌연변이를 갖는 인간화 ttr 좌위를 포함하는 비-인간 동물 및 사용 방법
JP2020530990A (ja) in vivoでの外因性ドナー核酸とのCRISPR/Cas誘導性組換えの評価
JP2022534560A (ja) ヒト化アルブミン遺伝子座を含む非ヒト動物
US20250040522A1 (en) Mutant myocilin disease model and uses thereof
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
Qi et al. Construction of a TAT-Cas9-EGFP Site-Specific Integration Eukaryotic Cell Line Using Efficient PEG10 Modification
US20230313173A1 (en) Systems and methods for identifying cells that have undergone genome editing
WO2025117934A1 (en) Novel compact crispr/cas12f1 system
KR20250171479A (ko) 세포 또는 유기체의 게놈으로의 DNA 서열의 표적화 혼입을 위한 Cas 9 레트로바이러스 인테그라제 시스템 및 Cas 9 재조합효소 시스템
WO2024181932A1 (en) Lambda integrase mutants and methods of their use
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