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 (58)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | 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 |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| 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 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| CA2956224A1 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| EP4434589A3 (en) | 2015-10-23 | 2025-05-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
| EP4036228A1 (en) | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| 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 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| WO2019165322A1 (en) * | 2018-02-22 | 2019-08-29 | Moore Tara | Crispr/cas9 systems, and methods of use thereof |
| CN109963945A (zh) * | 2016-08-20 | 2019-07-02 | 阿维利诺美国实验室股份有限公司 | 单一向导rna、crispr/cas9系统及其使用方法 |
| WO2020225754A1 (en) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| WO2018176009A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising 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 |
| KR20200037206A (ko) * | 2017-06-07 | 2020-04-08 | 도꾜 다이가꾸 | 과립상 각막 변성증에 대한 유전자 치료약 |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| KR20200121782A (ko) | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| 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 |
| CA3092497A1 (en) * | 2018-03-19 | 2019-09-26 | Crispr Therapeutics Ag | Novel rna-programmable endonuclease systems and uses thereof |
| EP3790961A1 (en) * | 2018-05-08 | 2021-03-17 | Katholieke Universiteit Leuven | Biosensor |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| 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 |
| 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. |
| 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 |
| WO2021117009A2 (en) * | 2019-12-11 | 2021-06-17 | Moore Tara | Allele-specific silencing of transforming growth factor beta induced gene with r124h mutation using short interfering rna |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | 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 |
| JP2025504931A (ja) * | 2022-01-26 | 2025-02-19 | オーソバイオ・セラピューティクス，インコーポレイテッド | 関節機能を改善する遺伝子編集 |
| US20250235559A1 (en) * | 2022-02-25 | 2025-07-24 | 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 |
| CN116286823A (zh) * | 2023-04-04 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 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 |
| PL4289948T3 (pl) * | 2012-05-25 | 2025-06-02 | The Regents Of The University Of California | Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji |
| PL2890780T3 (pl) | 2012-08-29 | 2021-02-08 | Sangamo Therapeutics, Inc. | Sposoby i kompozycje do leczenia zaburzeń genetycznych |
| CN105142669B (zh) * | 2012-12-06 | 2018-07-03 | 西格马-奥尔德里奇有限责任公司 | 基于crispr的基因组修饰和调控 |
| PL2784162T3 (pl) | 2012-12-12 | 2016-01-29 | Broad Inst Inc | Opracowanie systemów, metod oraz zoptymalizowanych kompozycji przewodnikowych do manipulacji sekwencyjnej |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| WO2014093622A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| CN105658796B (zh) | 2012-12-12 | 2021-10-26 | 布罗德研究所有限公司 | 用于序列操纵的crispr-cas组分系统、方法以及组合物 |
| PT2898075E (pt) | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências |
| CN105121648B (zh) | 2012-12-12 | 2021-05-07 | 布罗德研究所有限公司 | 用于序列操纵的系统、方法和优化的指导组合物的工程化 |
| JP6414945B2 (ja) * | 2013-03-11 | 2018-10-31 | Ｊｃｒファーマ株式会社 | ヒト角膜上皮シートの製造法 |
| CN116083487A (zh) | 2013-05-15 | 2023-05-09 | 桑格摩生物治疗股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| EP3674411A1 (en) * | 2013-06-17 | 2020-07-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| WO2015006290A1 (en) * | 2013-07-09 | 2015-01-15 | President And Fellows Of Harvard College | Multiplex rna-guided genome engineering |
| 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 |
| AU2016335572B2 (en) * | 2015-10-09 | 2022-12-08 | The Children's Hospital Of Philadelphia | Compositions and methods for treating Huntington's disease and related disorders |
| EP4036228A1 (en) * | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| CN109963945A (zh) * | 2016-08-20 | 2019-07-02 | 阿维利诺美国实验室股份有限公司 | 单一向导rna、crispr/cas9系统及其使用方法 |
-
2017
- 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 KR1020197007806A patent/KR102594051B1/ko active Active
- 2017-08-21 KR KR1020237036111A patent/KR20230155013A/ko not_active Ceased
- 2017-08-21 EP EP17844228.1A patent/EP3500677A4/en not_active Withdrawn
- 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
-
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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