JP2019524149A5 - - Google Patents
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- JP2019524149A5 JP2019524149A5 JP2019510339A JP2019510339A JP2019524149A5 JP 2019524149 A5 JP2019524149 A5 JP 2019524149A5 JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019524149 A5 JP2019524149 A5 JP 2019524149A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- seq
- crispr
- cas9 system
- snp
- 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
Links
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021214009A JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2024002942A JP2024041905A (ja) | 2016-08-20 | 2024-01-12 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662377586P | 2016-08-20 | 2016-08-20 | |
| US62/377,586 | 2016-08-20 | ||
| US201762462808P | 2017-02-23 | 2017-02-23 | |
| US62/462,808 | 2017-02-23 | ||
| US201762501750P | 2017-05-05 | 2017-05-05 | |
| US62/501,750 | 2017-05-05 | ||
| PCT/US2017/047861 WO2018039145A1 (en) | 2016-08-20 | 2017-08-21 | Single guide rna, crispr/cas9 systems, and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021214009A Division JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019524149A JP2019524149A (ja) | 2019-09-05 |
| JP2019524149A5 true JP2019524149A5 (enExample) | 2020-10-01 |
Family
ID=61246315
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019510339A Pending JP2019524149A (ja) | 2016-08-20 | 2017-08-21 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2021214009A Pending JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2024002942A Pending JP2024041905A (ja) | 2016-08-20 | 2024-01-12 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021214009A Pending JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2024002942A Pending JP2024041905A (ja) | 2016-08-20 | 2024-01-12 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20190185850A1 (enExample) |
| EP (1) | EP3500677A4 (enExample) |
| JP (3) | JP2019524149A (enExample) |
| KR (2) | KR102594051B1 (enExample) |
| CN (1) | CN109963945A (enExample) |
| WO (1) | WO2018039145A1 (enExample) |
Families Citing this family (59)
| 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 |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| 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 |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| EP4036228A1 (en) | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| US12214054B2 (en) | 2015-11-30 | 2025-02-04 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| JP7490211B2 (ja) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Ｃｐｆ１に基づくゲノム編集の治療適用 |
| 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 |
| WO2020225754A1 (en) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| EP3500677A4 (en) * | 2016-08-20 | 2020-04-01 | Avellino Lab USA, Inc. | SINGLE GUIDE RNA, CRISPR / CAS9 SYSTEMS AND METHOD FOR USE THEREOF |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use 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 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190123328A (ko) | 2017-03-09 | 2019-10-31 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 암 백신 |
| 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 |
| 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 |
| WO2018225807A1 (ja) * | 2017-06-07 | 2018-12-13 | 国立大学法人東京大学 | 顆粒状角膜変性症に対する遺伝子治療薬 |
| 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 |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| US20210032612A1 (en) * | 2018-02-22 | 2021-02-04 | Avellino Lab Usa, Inc. | CRISPR/Cas9 Systems, and Methods of Use Thereof |
| JP7550648B2 (ja) * | 2018-03-19 | 2024-09-13 | クリスパー セラピューティクス アーゲー | 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用 |
| CN112567030A (zh) * | 2018-05-08 | 2021-03-26 | 勒芬天主教大学 | 生物传感器 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| 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 |
| CA3121191A1 (en) | 2018-11-28 | 2020-06-04 | Crispr Therapeutics Ag | Optimized mrna encoding cas9 for use in lnps |
| 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 |
| TWI887234B (zh) | 2019-04-14 | 2025-06-21 | 美國公爵大學 | 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除 |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN110499335B (zh) * | 2019-08-08 | 2023-03-28 | 复旦大学 | CRISPR/SauriCas9基因编辑系统及其应用 |
| CN110551763B (zh) * | 2019-08-08 | 2023-03-10 | 复旦大学 | CRISPR/SlutCas9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 |
| EP4073095A4 (en) * | 2019-12-11 | 2024-11-27 | Avellino Lab USA, Inc. | Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna |
| 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 |
| CN111926015B (zh) * | 2020-08-24 | 2022-07-15 | 武汉纽福斯生物科技有限公司 | 寡核苷酸、病毒载体及其应用和RNAi药物制剂 |
| AU2022216614B2 (en) | 2021-02-05 | 2026-03-05 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| WO2023034704A1 (en) * | 2021-08-31 | 2023-03-09 | The Regents Of The University Of California | Products and methods for annotating gene function using locally haploid, human non-cancer cells |
| EP4469577A4 (en) * | 2022-01-26 | 2026-04-15 | Orthobio Therapeutics Inc | GENE EDITING TO IMPROVE JOINT FUNCTION |
| WO2023161873A1 (en) * | 2022-02-25 | 2023-08-31 | Incisive Genetics, Inc. | Gene editing reporter system and guide rna and composition related thereto; composition and method for knocking out dna with more than two grnas; gene editing in the eye; and gene editing using base editors |
| CN115948537B (zh) * | 2022-12-19 | 2024-04-09 | 湖南家辉生物技术有限公司 | 一种基因chst3复合杂合突变的应用及检测试剂和应用 |
| CN116286823B (zh) * | 2023-04-04 | 2026-06-19 | 上海尧唐生物科技股份有限公司 | 靶向TGFBI基因的sgRNA、载体及其应用 |
| WO2025043140A1 (en) * | 2023-08-23 | 2025-02-27 | Emendobio Inc. | Methods and compositions for modifying expression of a mutant transforming growth factor beta induced (tgfbi) allele |
| CN119464347B (zh) * | 2024-11-26 | 2025-10-03 | 扬州大学 | 基于CRISPR-Cas9系统的噬菌体基因组编辑载体及其编辑方法和应用 |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US9895451B2 (en) | 2011-12-02 | 2018-02-20 | Yale University | Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof |
| ES2728782T3 (es) * | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| EP2890780B8 (en) | 2012-08-29 | 2020-08-19 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| DK3363902T3 (da) * | 2012-12-06 | 2020-01-02 | Sigma Aldrich Co Llc | Crispr-baseret genommodifikation og -regulering |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| EP3064585B1 (en) | 2012-12-12 | 2020-02-05 | The Broad Institute, Inc. | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| KR102955556B1 (ko) * | 2012-12-12 | 2026-04-22 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물 |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| PT2931898E (pt) | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| MX380562B (es) | 2012-12-12 | 2025-03-12 | Broad Inst Inc | Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias. |
| EP2975117B1 (en) * | 2013-03-11 | 2018-01-10 | JCR Pharmaceuticals Co., Ltd. | Method for producing human corneal epithelium sheet |
| EP2997146A4 (en) | 2013-05-15 | 2017-04-26 | Sangamo BioSciences, Inc. | Methods and compositions for treatment of a genetic condition |
| ES2777217T3 (es) * | 2013-06-17 | 2020-08-04 | Broad Inst Inc | Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias |
| KR20260047646A (ko) * | 2013-07-09 | 2026-04-08 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 멀티플렉스 rna-가이드된 게놈 조작 |
| US20150044772A1 (en) * | 2013-08-09 | 2015-02-12 | Sage Labs, Inc. | Crispr/cas system-based novel fusion protein and its applications in genome editing |
| US10787684B2 (en) * | 2013-11-19 | 2020-09-29 | President And Fellows Of Harvard College | Large gene excision and insertion |
| WO2016049024A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo |
| EP3359662A4 (en) * | 2015-10-09 | 2019-06-19 | The Children's Hospital of Philadelphia | COMPOSITIONS AND METHODS OF TREATING HUNTINGTON DISEASE AND RELATED DISEASES |
| EP4036228A1 (en) * | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| EP3500677A4 (en) * | 2016-08-20 | 2020-04-01 | Avellino Lab USA, Inc. | SINGLE GUIDE RNA, CRISPR / CAS9 SYSTEMS AND METHOD FOR USE THEREOF |
-
2017
- 2017-08-21 EP EP17844228.1A patent/EP3500677A4/en not_active Withdrawn
- 2017-08-21 KR KR1020197007806A patent/KR102594051B1/ko active Active
- 2017-08-21 US US16/326,908 patent/US20190185850A1/en not_active Abandoned
- 2017-08-21 WO PCT/US2017/047861 patent/WO2018039145A1/en not_active Ceased
- 2017-08-21 CN CN201780064151.5A patent/CN109963945A/zh active Pending
- 2017-08-21 JP JP2019510339A patent/JP2019524149A/ja active Pending
- 2017-08-21 KR KR1020237036111A patent/KR20230155013A/ko not_active Ceased
-
2021
- 2021-12-28 JP JP2021214009A patent/JP2022046694A/ja active Pending
-
2024
- 2024-01-12 JP JP2024002942A patent/JP2024041905A/ja active Pending
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