BR112019016765A2 - Molécula de cas9 modificada isolada, método para produzir uma cas9 modificada, complexos de proteína ou ribonucleoproteína ou complexos de proteína e ribonucleoproteína misturadas ou lipídeo, proteína de fusão, sequência de nucleotídeos, ácido nucleico, vetor, célula, animal ou planta, composição farmacêutica, uso in vitro de uma cas9 modificada recombinante, kit de partes, e, método in vitro para alterar o genoma de uma célula - Google Patents
Molécula de cas9 modificada isolada, método para produzir uma cas9 modificada, complexos de proteína ou ribonucleoproteína ou complexos de proteína e ribonucleoproteína misturadas ou lipídeo, proteína de fusão, sequência de nucleotídeos, ácido nucleico, vetor, célula, animal ou planta, composição farmacêutica, uso in vitro de uma cas9 modificada recombinante, kit de partes, e, método in vitro para alterar o genoma de uma célula Download PDFInfo
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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- C07K—PEPTIDES
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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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- 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
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Applications Claiming Priority (3)
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|---|---|---|---|
| IT102017000016321 | 2017-02-14 | ||
| IT102017000016321A IT201700016321A1 (it) | 2017-02-14 | 2017-02-14 | Mutanti di cas9 ad alta specificita' e loro applicazioni. |
| PCT/EP2018/053717 WO2018149888A1 (en) | 2017-02-14 | 2018-02-14 | High-fidelity cas9 variants and applications thereof |
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| BR112019016765A2 true BR112019016765A2 (pt) | 2020-05-26 |
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| WO2020257325A1 (en) * | 2019-06-17 | 2020-12-24 | Vertex Pharmaceuticals Inc. | Compositions and methods for editing beta-globin for treatment of hemaglobinopathies |
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| JP7680440B2 (ja) * | 2019-10-15 | 2025-05-20 | エージェンシー フォー サイエンス， テクノロジー アンド リサーチ | 核酸修飾酵素活性を測定するためのアッセイ法 |
| WO2021122944A1 (en) | 2019-12-18 | 2021-06-24 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| AU2021236230B2 (en) * | 2020-03-11 | 2024-09-26 | Sigma-Aldrich Co. Llc | High fidelity SpCas9 nucleases for genome modification |
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| WO2022018638A1 (en) | 2020-07-21 | 2022-01-27 | Crispr Therapeutics Ag | Genome-editing compositions and methods to modulate faah for treatment of neurological disorders |
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| CN112680430B (zh) * | 2020-12-28 | 2023-06-06 | 南方医科大学 | 一种CRISPR SpCas9突变体及其应用 |
| CN113005141A (zh) * | 2021-01-05 | 2021-06-22 | 温州医科大学 | 高活性突变体构成的基因编辑工具及制备方法和修复先天性视网膜劈裂症致病基因的方法 |
| WO2022152746A1 (en) | 2021-01-13 | 2022-07-21 | Alia Therapeutics Srl | K526d cas9 variants and applications thereof |
| CN112626070A (zh) * | 2021-01-13 | 2021-04-09 | 海南微氪生物科技股份有限公司 | 一种基于切离酶基因编辑技术的定向修复系统 |
| WO2022238958A1 (en) | 2021-05-12 | 2022-11-17 | Crispr Therapeutics Ag | Multiplex gene editing |
| WO2023285431A1 (en) | 2021-07-12 | 2023-01-19 | Alia Therapeutics Srl | Compositions and methods for allele specific treatment of retinitis pigmentosa |
| US20240382528A1 (en) * | 2021-10-05 | 2024-11-21 | The Board Of Trustees Of The Leland Stanford Junior University | Treatment of polycythemia vera via crispr/aav6 genome editing |
| WO2025087413A1 (zh) * | 2023-10-25 | 2025-05-01 | 上海津曼特生物科技有限公司 | 靶向ttr的基因编辑组合物 |
| CN117887760A (zh) * | 2024-01-22 | 2024-04-16 | 中国医学科学院北京协和医院 | SQSTM1/p62基因新致病突变c.1143delC的敲入小鼠模型及其构建方法 |
| CN118497204B (zh) * | 2024-07-16 | 2024-12-03 | 西北农林科技大学深圳研究院 | 一种质粒CRISPR-pCas9n及基因编辑的方法与应用 |
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| US11685935B2 (en) * | 2013-05-29 | 2023-06-27 | Cellectis | Compact scaffold of Cas9 in the type II CRISPR system |
| US11180792B2 (en) * | 2015-01-28 | 2021-11-23 | The Regents Of The University Of California | Methods and compositions for labeling a single-stranded target nucleic acid |
| US9944912B2 (en) | 2015-03-03 | 2018-04-17 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| US11214779B2 (en) * | 2015-04-08 | 2022-01-04 | University of Pittsburgh—of the Commonwealth System of Higher Education | Activatable CRISPR/CAS9 for spatial and temporal control of genome editing |
| KR102840885B1 (ko) * | 2015-06-18 | 2025-07-30 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적외 효과를 감소시키는 crispr 효소 돌연변이 |
| US9512446B1 (en) * | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| IT201700016321A1 (it) | 2017-02-14 | 2018-08-14 | Univ Degli Studi Di Trento | Mutanti di cas9 ad alta specificita' e loro applicazioni. |
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| AU2018221644A1 (en) | 2019-08-29 |
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| EP3583209A1 (en) | 2019-12-25 |
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| KR20190116407A (ko) | 2019-10-14 |
| US11525127B2 (en) | 2022-12-13 |
| EP3583209C0 (en) | 2024-12-04 |
| CN110520528A (zh) | 2019-11-29 |
| EP3583209B1 (en) | 2024-12-04 |
| US20200149020A1 (en) | 2020-05-14 |
| EP3583209B8 (en) | 2025-02-26 |
| EA037825B1 (ru) | 2021-05-25 |
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