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
- 108091033409 CRISPR Proteins 0.000 claims 125
- 238000010354 CRISPR gene editing Methods 0.000 claims 56
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- 206010011005 corneal dystrophy Diseases 0.000 claims 26
- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 claims 20
- 108091027544 Subgenomic mRNA Proteins 0.000 claims 18
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- 102220540600 UbiA prenyltransferase domain-containing protein 1_L188H_mutation Human genes 0.000 claims 1
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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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3500677A4 (OSRAM) |
| JP (3) | JP2019524149A (OSRAM) |
| KR (2) | KR20230155013A (OSRAM) |
| CN (1) | CN109963945A (OSRAM) |
| WO (1) | WO2018039145A1 (OSRAM) |
Families Citing this family (56)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | 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 |
| 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 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| JP6929865B2 (ja) | 2015-11-13 | 2021-09-01 | タラ ムーア | 角膜ジストロフィーの治療方法 |
| EP3384055B1 (en) | 2015-11-30 | 2025-07-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP3487523B1 (en) | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
| GB2568182A (en) | 2016-08-03 | 2019-05-08 | Harvard College | Adenosine nucleobase editors and uses thereof |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| US20190185850A1 (en) * | 2016-08-20 | 2019-06-20 | Avellino Lab Usa, Inc. | Single guide rna/crispr/cas9 systems, and methods of use thereof |
| WO2020225754A1 (en) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| GB2573062A (en) | 2016-10-14 | 2019-10-23 | 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 |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | 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 |
| US11268082B2 (en) | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| EP3636754A4 (en) * | 2017-06-07 | 2021-03-17 | The University Of Tokyo | GENE THERAPY FOR GRANULAR CORNEAL DYSTROPHY |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | 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 |
| US20210032612A1 (en) * | 2018-02-22 | 2021-02-04 | Avellino Lab Usa, Inc. | CRISPR/Cas9 Systems, and Methods of Use Thereof |
| CA3092497A1 (en) | 2018-03-19 | 2019-09-26 | Crispr Therapeutics Ag | Novel rna-programmable endonuclease systems and uses thereof |
| US20210230690A1 (en) * | 2018-05-08 | 2021-07-29 | Katholieke Universiteit Leuven | Biosensor |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| JP7564102B2 (ja) | 2018-11-28 | 2024-10-08 | クリスパー セラピューティクス アクチェンゲゼルシャフト | ＬＮＰでの使用に最適化された、ＣＡＳ９をコードするｍＲＮＡ |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| AU2020240109A1 (en) | 2019-03-19 | 2021-09-30 | President And Fellows Of Harvard College | 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 |
| CN110551763B (zh) * | 2019-08-08 | 2023-03-10 | 复旦大学 | CRISPR/SlutCas9基因编辑系统及其应用 |
| CN110499335B (zh) * | 2019-08-08 | 2023-03-28 | 复旦大学 | CRISPR/SauriCas9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 |
| CN115515968A (zh) * | 2019-12-11 | 2022-12-23 | 阿维利诺美国实验室股份有限公司 | 使用短干扰RNA对具有R124H突变的转化生长因子β诱导基因的等位基因特异性沉默 |
| BR112022022603A2 (pt) | 2020-05-08 | 2023-01-17 | Broad Inst Inc | Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla |
| 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 |
| US12171813B2 (en) | 2021-02-05 | 2024-12-24 | 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 |
| EP4469577A2 (en) * | 2022-01-26 | 2024-12-04 | Orthobio Therapeutics, Inc. | Gene editing to improve joint function |
| CA3244722A1 (en) * | 2022-02-25 | 2023-08-31 | Incisive Genetics, Inc. | Gene editing reporter system and RNA guide and associated composition; composition and method for removing DNA with more than two RNAs; gene editing in the eye; and gene editing using database 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 |
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| 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 |
| 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 |
| 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 |
| 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 |
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-
2017
- 2017-08-21 US US16/326,908 patent/US20190185850A1/en not_active Abandoned
- 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 CN CN201780064151.5A patent/CN109963945A/zh active Pending
- 2017-08-21 WO PCT/US2017/047861 patent/WO2018039145A1/en not_active Ceased
- 2017-08-21 JP JP2019510339A patent/JP2019524149A/ja active Pending
- 2017-08-21 KR KR1020197007806A patent/KR102594051B1/ko active Active
-
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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