JP2020513814A - Crispr cas9免疫ステルス(immune stealth)の操作 - Google Patents

Crispr cas9免疫ステルス(immune stealth)の操作 Download PDF

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JP2020513814A
JP2020513814A JP2019550861A JP2019550861A JP2020513814A JP 2020513814 A JP2020513814 A JP 2020513814A JP 2019550861 A JP2019550861 A JP 2019550861A JP 2019550861 A JP2019550861 A JP 2019550861A JP 2020513814 A JP2020513814 A JP 2020513814A
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cas9
protein
proteins
aav
mhc
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JP2020513814A5 (enExample
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マリ,プラシャント
モレノ コリャド,アナ
モレノ コリャド,アナ
パーマー,ネイサン
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University of California
University of California Berkeley
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University of California San Diego UCSD
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    • CCHEMISTRY; METALLURGY
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    • 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]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/0004Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/12Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from bacteria
    • C07K16/1267Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from bacteria from Gram-positive bacteria
    • C07K16/1275Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from bacteria from Gram-positive bacteria from Streptococcus (G)
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    • 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
    • C12N15/11DNA 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
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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
    • C12N15/63Introduction 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
    • C12N15/86Viral vectors
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
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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
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Diabetes (AREA)
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JP2019550861A 2017-03-14 2018-03-13 Crispr cas9免疫ステルス(immune stealth)の操作 Pending JP2020513814A (ja)

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 (2)

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JP2020513814A true JP2020513814A (ja) 2020-05-21
JP2020513814A5 JP2020513814A5 (enExample) 2021-09-02

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

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI 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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin 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
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
WO2017053879A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 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
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018201086A1 (en) 2017-04-28 2018-11-01 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
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
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
SG11202009452WA (en) * 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
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
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110499334A (zh) * 2019-08-08 2019-11-26 复旦大学 CRISPR/SlugCas9基因编辑系统及其应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
IL298134A (en) * 2020-05-12 2023-01-01 Lonza Houston Inc Methods and kits for the detection of adeno-associated viruses
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
WO2024112479A1 (en) * 2022-11-22 2024-05-30 The Regents Of The University Of California Crispr-cas effector polypeptides, lipid nanoparticles, and methods of use thereof
WO2024211456A1 (en) * 2023-04-03 2024-10-10 The Broad Institute, Inc. Engineered type ii cas polynucleotides with reduced immunogenicity and uses thereof
CN117448300B (zh) * 2023-05-08 2024-04-30 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
US20250001010A1 (en) 2023-06-30 2025-01-02 Christiana Care Gene Editing Institute, Inc. Nras gene knockout for treatment of cancer
US20250319206A1 (en) 2024-04-04 2025-10-16 Christina Care Gene Editing Institute, Inc. Crispr/cas gene editing of neh4 and/or neh5 domains in nrf2
WO2025226816A1 (en) 2024-04-23 2025-10-30 Christiana Care Gene Editing Institute, Inc. Methods of identifying and correlating crispr-induced exon skipping to phenotypic outcomes

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015523856A (ja) * 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Rna依存性標的dna修飾およびrna依存性転写調節のための方法および組成物
JP2016521995A (ja) * 2013-06-17 2016-07-28 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのCRISPR−Cas系および組成物の送達、使用および治療上の適用
JP2016523560A (ja) * 2013-07-10 2016-08-12 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 Rna誘導性遺伝子制御および編集のための直交性cas9タンパク質

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6730507B1 (en) 1993-06-24 2004-05-04 Merck & Co., Inc. Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration
CA2438628A1 (en) * 2001-02-19 2002-08-29 Merck Patent Gesellschaft Mit Beschraenkter Haftung Artificial proteins with reduced immunogenicity
RU2003130060A (ru) * 2001-03-20 2005-04-10 Мерк Патент ГмбХ (DE) Модифицированный инсулин с уменьшенной иммуногенностью
MXPA04000339A (es) * 2001-07-13 2004-05-04 Merck Patent Gesellscahft Mit MeTODOS PARA REDUCIR INMUNOGENICIDAD DE POLIPePTIDOS.
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
CN105247044B (zh) * 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
EP3498845B1 (en) * 2014-04-01 2022-06-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
NZ728437A (en) * 2014-08-27 2018-02-23 Caribou Biosciences Inc Methods for increasing cas9-mediated engineering efficiency

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015523856A (ja) * 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Rna依存性標的dna修飾およびrna依存性転写調節のための方法および組成物
JP2016521995A (ja) * 2013-06-17 2016-07-28 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのCRISPR−Cas系および組成物の送達、使用および治療上の適用
JP2016523560A (ja) * 2013-07-10 2016-08-12 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 Rna誘導性遺伝子制御および編集のための直交性cas9タンパク質

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
NAT. METHODS (2016) VOL.13, NO.10, P.868-874, SUPPLEMENTARY INFORMATION, JPN6022009934, ISSN: 0004727222 *
NUCLEIC ACIDS RES. (2014) VO.42, NO.4, P.2577-2590, SUPPLEMENTARY DATA, JPN6022009931, ISSN: 0004892835 *

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EP3595694A1 (en) 2020-01-22
US20200299657A1 (en) 2020-09-24
IL268829A (en) 2019-10-31
EP3595694A4 (en) 2021-06-09
US11332727B2 (en) 2022-05-17
GB2574561A (en) 2019-12-11
GB201913821D0 (en) 2019-11-06
CN110913881A (zh) 2020-03-24
WO2018170015A1 (en) 2018-09-20

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