CN110913881A - 工程化crispr cas9免疫隐身 - Google Patents

工程化crispr cas9免疫隐身 Download PDF

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Publication number
CN110913881A
CN110913881A CN201880031835.XA CN201880031835A CN110913881A CN 110913881 A CN110913881 A CN 110913881A CN 201880031835 A CN201880031835 A CN 201880031835A CN 110913881 A CN110913881 A CN 110913881A
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cas9
protein
aav
proteins
mhc
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CN201880031835.XA
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Chinese (zh)
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普拉尚特·马利
阿纳·莫雷诺·科拉多
内森·帕尔默
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University of California San Diego UCSD
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University of California San Diego UCSD
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/12—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from bacteria
    • C07K16/1267—Gram-positive bacteria
    • C07K16/1275—Streptococcus (G)
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Animal Behavior & Ethology (AREA)
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  • Physics & Mathematics (AREA)
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CN201880031835.XA 2017-03-14 2018-03-13 工程化crispr cas9免疫隐身 Pending CN110913881A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762471267P 2017-03-14 2017-03-14
US62/471,267 2017-03-14
US201862614875P 2018-01-08 2018-01-08
US62/614,875 2018-01-08
PCT/US2018/022258 WO2018170015A1 (en) 2017-03-14 2018-03-13 Engineering crispr cas9 immune stealth

Publications (1)

Publication Number Publication Date
CN110913881A true CN110913881A (zh) 2020-03-24

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US (1) US11332727B2 (https=)
EP (1) EP3595694A4 (https=)
JP (1) JP2020513814A (https=)
CN (1) CN110913881A (https=)
GB (1) GB2574561A (https=)
IL (1) IL268829A (https=)
WO (1) WO2018170015A1 (https=)

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CN113717960A (zh) * 2021-08-27 2021-11-30 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
CN116751764A (zh) * 2023-05-08 2023-09-15 珠海舒桐医疗科技有限公司 一种Cas9蛋白、II型CRISPR/Cas9基因编辑系统及应用
WO2025190256A1 (en) * 2024-03-11 2025-09-18 Geneditbio Limited Type ii cas protein and uses thereof
TWI910162B (zh) * 2020-05-05 2026-01-01 美國公爵大學 跨物種相容腺相關病毒組合物及其使用方法

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US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
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US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
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CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
CN108026526B (zh) 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
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CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
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US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
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KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI910162B (zh) * 2020-05-05 2026-01-01 美國公爵大學 跨物種相容腺相關病毒組合物及其使用方法
US12577586B2 (en) 2020-05-05 2026-03-17 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
CN113717960A (zh) * 2021-08-27 2021-11-30 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
CN116751764A (zh) * 2023-05-08 2023-09-15 珠海舒桐医疗科技有限公司 一种Cas9蛋白、II型CRISPR/Cas9基因编辑系统及应用
CN116751764B (zh) * 2023-05-08 2024-01-30 珠海舒桐医疗科技有限公司 一种Cas9蛋白、II型CRISPR/Cas9基因编辑系统及应用
WO2025190256A1 (en) * 2024-03-11 2025-09-18 Geneditbio Limited Type ii cas protein and uses thereof

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IL268829A (en) 2019-10-31
EP3595694A1 (en) 2020-01-22
GB2574561A (en) 2019-12-11
US20200299657A1 (en) 2020-09-24
US11332727B2 (en) 2022-05-17
GB201913821D0 (en) 2019-11-06
JP2020513814A (ja) 2020-05-21
EP3595694A4 (en) 2021-06-09
WO2018170015A1 (en) 2018-09-20

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