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 PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- cas9
- protein
- proteins
- aav
- mhc
- 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
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Classifications
-
- 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
- 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]
-
- 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
- C07K16/08—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from viruses
-
- 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)
-
- 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
- C12N15/11—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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Urology & Nephrology (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Cell Biology (AREA)
- Endocrinology (AREA)
- Rheumatology (AREA)
- Toxicology (AREA)
- Analytical Chemistry (AREA)
- Food Science & Technology (AREA)
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)
| Publication Number | Publication Date |
|---|---|
| JP2020513814A true JP2020513814A (ja) | 2020-05-21 |
| JP2020513814A5 JP2020513814A5 (enExample) | 2021-09-02 |
Family
ID=63522593
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019550861A Pending JP2020513814A (ja) | 2017-03-14 | 2018-03-13 | Crispr cas9免疫ステルス(immune stealth)の操作 |
Country Status (7)
| Country | Link |
|---|---|
| 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 (56)
| 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 |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| 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 |
| WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | 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 |
| US11911415B2 (en) | 2015-06-09 | 2024-02-27 | 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 |
| WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| WO2017165826A1 (en) | 2016-03-25 | 2017-09-28 | 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 |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | 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 |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| EP3555297A1 (en) | 2016-12-19 | 2019-10-23 | 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 |
| WO2018129368A2 (en) | 2017-01-06 | 2018-07-12 | Editas Medicine, Inc. | Methods of assessing nuclease cleavage |
| KR20190123328A (ko) | 2017-03-09 | 2019-10-31 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 암 백신 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | 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 |
| CN110997908A (zh) | 2017-06-09 | 2020-04-10 | 爱迪塔斯医药公司 | 工程化的cas9核酸酶 |
| 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 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base 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 |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| BR112020020266A2 (pt) * | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020191241A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | 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 |
| 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 |
| CA3177481A1 (en) | 2020-05-08 | 2021-11-11 | David R. Liu | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| CN115335704A (zh) * | 2020-05-12 | 2022-11-11 | 隆萨休斯敦股份有限公司 | 用于检测腺相关病毒的方法和试剂盒 |
| 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 |
| US20260185070A1 (en) * | 2022-11-22 | 2026-07-02 | 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 |
| AU2024309386A1 (en) | 2023-06-30 | 2025-12-18 | Christiana Care Gene Editing Institute, Inc. | Nras gene knockout for treatment of cancer |
| WO2025190256A1 (en) * | 2024-03-11 | 2025-09-18 | Geneditbio Limited | Type ii cas protein and uses thereof |
| WO2025213016A2 (en) | 2024-04-04 | 2025-10-09 | Christiana 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)
| 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)
| 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 |
| PL362324A1 (en) * | 2001-02-19 | 2004-10-18 | Merck Patent Gmbh | Artificial fusion proteins with reduced immunogenicity |
| CA2441388A1 (en) * | 2001-03-20 | 2002-09-26 | Merck Patent Gesellschaft Mit Beschraenkter Haftung | Modified insulin with reduced immunogenicity |
| CA2453406A1 (en) * | 2001-07-13 | 2003-01-23 | Merck Patent Gesellschaft Mit Beschraenkter Haftung | Methods for reducing immunogenicity of polypeptides |
| 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 |
| US11136557B2 (en) * | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| WO2015153789A1 (en) * | 2014-04-01 | 2015-10-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1) |
| ES2730378T3 (es) * | 2014-08-27 | 2019-11-11 | Caribou Biosciences Inc | Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9 |
-
2018
- 2018-03-13 GB GB1913821.3A patent/GB2574561A/en not_active Withdrawn
- 2018-03-13 WO PCT/US2018/022258 patent/WO2018170015A1/en not_active Ceased
- 2018-03-13 US US16/494,269 patent/US11332727B2/en active Active
- 2018-03-13 CN CN201880031835.XA patent/CN110913881A/zh active Pending
- 2018-03-13 EP EP18768009.5A patent/EP3595694A4/en not_active Withdrawn
- 2018-03-13 JP JP2019550861A patent/JP2020513814A/ja active Pending
-
2019
- 2019-08-21 IL IL26882919A patent/IL268829A/en unknown
Patent Citations (3)
| 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)
| 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 * |
Also Published As
| Publication number | Publication date |
|---|---|
| US11332727B2 (en) | 2022-05-17 |
| CN110913881A (zh) | 2020-03-24 |
| WO2018170015A1 (en) | 2018-09-20 |
| GB2574561A (en) | 2019-12-11 |
| EP3595694A4 (en) | 2021-06-09 |
| US20200299657A1 (en) | 2020-09-24 |
| EP3595694A1 (en) | 2020-01-22 |
| IL268829A (en) | 2019-10-31 |
| GB201913821D0 (en) | 2019-11-06 |
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