BR112020010479A2 - sistemas cas9 geneticamente modificados para modificação de genoma eucariótico - Google Patents

sistemas cas9 geneticamente modificados para modificação de genoma eucariótico Download PDF

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Publication number
BR112020010479A2
BR112020010479A2 BR112020010479-5A BR112020010479A BR112020010479A2 BR 112020010479 A2 BR112020010479 A2 BR 112020010479A2 BR 112020010479 A BR112020010479 A BR 112020010479A BR 112020010479 A2 BR112020010479 A2 BR 112020010479A2
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Brazil
Prior art keywords
genetically modified
sequence
protein
fact
cell
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BR112020010479-5A
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English (en)
Portuguese (pt)
Inventor
Timothy Seebeck
Fuqiang Chen
Gregory D. Davis
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Sigma-Aldrich Co. Llc
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Publication of BR112020010479A2 publication Critical patent/BR112020010479A2/pt

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    • 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
    • C12N9/14Hydrolases (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
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    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing 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
    • 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
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/85Fusion polypeptide containing an RNA binding domain
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/21Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21001Deoxyribonuclease I (3.1.21.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
BR112020010479-5A 2018-02-15 2019-02-15 sistemas cas9 geneticamente modificados para modificação de genoma eucariótico BR112020010479A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862631304P 2018-02-15 2018-02-15
US62/631,304 2018-02-15
US201862720525P 2018-08-21 2018-08-21
US62/720,525 2018-08-21
PCT/US2019/018335 WO2019161290A1 (en) 2018-02-15 2019-02-15 Engineered cas9 systems for eukaryotic genome modification

Publications (1)

Publication Number Publication Date
BR112020010479A2 true BR112020010479A2 (pt) 2020-11-24

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BR112020010479-5A BR112020010479A2 (pt) 2018-02-15 2019-02-15 sistemas cas9 geneticamente modificados para modificação de genoma eucariótico

Country Status (11)

Country Link
US (2) US10767193B2 (https=)
EP (1) EP3752607A1 (https=)
JP (3) JP7109547B2 (https=)
KR (3) KR102465067B1 (https=)
CN (1) CN111902536B (https=)
AU (3) AU2019222568B2 (https=)
BR (1) BR112020010479A2 (https=)
CA (2) CA3281020A1 (https=)
IL (2) IL274528B2 (https=)
SG (1) SG11202007382TA (https=)
WO (1) WO2019161290A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US11111492B2 (en) * 2017-03-06 2021-09-07 Florida State University Research Foundation, Inc. Genome engineering methods using a cytosine-specific Cas9
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US10913941B2 (en) 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
US10947517B2 (en) 2019-02-15 2021-03-16 Sigma-Aldrich Co. Llc CRISPR/Cas fusion proteins and systems
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
IL292517A (en) 2019-12-17 2022-06-01 Sigma Aldrich Co Llc Genome editing in Bacteroidetes
GB2594339B (en) * 2020-02-14 2023-02-08 Metagenomi Inc Enzymes with RUVC domains
US20230058352A1 (en) * 2020-03-11 2023-02-23 Sigma-Aldrich Co. Llc High Fidelity SpCas9 Nucleases for Genome Modification
WO2021202568A1 (en) 2020-03-31 2021-10-07 Metagenomi Ip Technologies, Llc Class ii, type ii crispr systems
CN112159801B (zh) * 2020-07-13 2022-11-15 复旦大学 SlugCas9-HF蛋白、含有SlugCas9-HF蛋白的基因编辑系统及应用
KR102679001B1 (ko) * 2020-09-21 2024-06-28 고려대학교 산학협력단 신규의 개량된 염기 편집 또는 교정용 융합단백질 및 이의 용도
US20220145334A1 (en) * 2020-11-06 2022-05-12 Pairwise Plants Services, Inc. Compositions and methods for rna-encoded dna-replacement of alleles
CN113717960B (zh) * 2021-08-27 2023-07-18 电子科技大学 一种新Cas9蛋白、CRISPR-Cas9基因组定向编辑载体及基因组编辑方法
AU2022335499A1 (en) 2021-08-27 2024-02-22 Metagenomi Therapeutics, Inc. Enzymes with ruvc domains
AU2022396533A1 (en) 2021-11-24 2024-05-02 Metagenomi Therapeutics, Inc. Endonuclease systems
CN114657181B (zh) * 2022-04-01 2023-08-25 安徽大学 一种靶向H1.4的sgRNA以及H1.4基因编辑方法
EP4592322A1 (en) * 2022-09-23 2025-07-30 Chuang, Woei-jer Fusion protein targeting intergrin alpha(iib)beta3 and containing tissue plasminogen activator or variant thereof and use thereof

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MXPA04005010A (es) 2001-11-26 2005-04-08 Advanced Cell Tech Inc METODO PARA HACER Y USAR NUCLEOS CELULARES SOMáTICOS METODOS PARA HACER Y USAR NUCLEOS CELULARES SOMáTICOS HUMANOS REPROGRAMADOS Y CELULAS DEL TALLO HUMANAS AUTOLOGAS E ISOGENICAS.
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
EP2840140B2 (en) * 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
US9074199B1 (en) * 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
JP2017503514A (ja) * 2014-01-24 2017-02-02 ノースカロライナ ステート ユニバーシティーNorth Carolina State University Cas9ターゲッティングをガイドする配列に関する方法および組成物
EP3122880B1 (en) * 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3186375A4 (en) * 2014-08-28 2019-03-13 North Carolina State University Novel CAS9 PROTEINS AND CHARACTERISTICS FOR DNA TARGETING AND GENOME EDITING
EP3201340B1 (en) * 2014-10-01 2020-12-02 The General Hospital Corporation Methods for increasing efficiency of nuclease-induced homology-directed repair
CA3209273A1 (en) * 2016-06-02 2017-12-07 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
JP7305534B2 (ja) 2016-07-06 2023-07-10 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 疼痛関連障害を処置するための物質及び方法
BR112019012155A2 (pt) * 2016-12-14 2019-11-12 Wageningen Universiteit uso de pelo menos uma molécula-guia de rna e uma proteína cas, método de ligação, clivagem, marcação ou modificação de um polinucleotídeo alvo de fita dupla, célula transformada, e, complexo de nucleoproteína
KR102655021B1 (ko) * 2017-07-11 2024-04-04 시그마-알드리치 컴퍼니., 엘엘씨 표적된 게놈 변형을 개선하기 위한 뉴클레오솜 상호작용 단백질 도메인 사용
SG11202000840YA (en) * 2017-07-31 2020-02-27 Reflection Biotechnologies Ltd Cellular models of and therapies for ocular diseases

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Publication number Publication date
KR102465067B1 (ko) 2022-11-10
WO2019161290A1 (en) 2019-08-22
US20190249200A1 (en) 2019-08-15
AU2022200130A1 (en) 2022-02-10
US12297449B2 (en) 2025-05-13
KR20220015502A (ko) 2022-02-08
JP2024161376A (ja) 2024-11-19
AU2024202158A1 (en) 2024-05-02
AU2019222568B2 (en) 2021-10-14
CA3084020A1 (en) 2019-08-22
IL274528B2 (en) 2025-10-01
KR20230022258A (ko) 2023-02-14
US10767193B2 (en) 2020-09-08
IL274528A (en) 2020-06-30
EP3752607A1 (en) 2020-12-23
JP2021505180A (ja) 2021-02-18
AU2022200130B2 (en) 2024-03-07
CA3281020A1 (en) 2026-03-02
KR102494449B1 (ko) 2023-01-31
KR20200121342A (ko) 2020-10-23
CN111902536B (zh) 2024-07-30
JP7109547B2 (ja) 2022-07-29
US20200354752A1 (en) 2020-11-12
CA3084020C (en) 2025-11-25
JP2022122910A (ja) 2022-08-23
JP7559005B2 (ja) 2024-10-01
SG11202007382TA (en) 2020-08-28
AU2019222568A1 (en) 2020-05-21
KR102684890B1 (ko) 2024-07-12
IL321024A (en) 2025-07-01
IL274528B1 (en) 2025-06-01
CN111902536A (zh) 2020-11-06

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