CN112567036A - 用于基因编辑的修饰的向导rna - Google Patents
用于基因编辑的修饰的向导rna Download PDFInfo
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- CN112567036A CN112567036A CN201980052704.4A CN201980052704A CN112567036A CN 112567036 A CN112567036 A CN 112567036A CN 201980052704 A CN201980052704 A CN 201980052704A CN 112567036 A CN112567036 A CN 112567036A
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12N2320/50—Methods for regulating/modulating their activity
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- Crystallography & Structural Chemistry (AREA)
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- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Mycology (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862682838P | 2018-06-08 | 2018-06-08 | |
| US201862682820P | 2018-06-08 | 2018-06-08 | |
| US62/682,820 | 2018-06-08 | ||
| US62/682,838 | 2018-06-08 | ||
| PCT/US2019/036160 WO2019237069A1 (en) | 2018-06-08 | 2019-06-07 | Modified guide rnas for gene editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112567036A true CN112567036A (zh) | 2021-03-26 |
Family
ID=68770688
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980052704.4A Pending CN112567036A (zh) | 2018-06-08 | 2019-06-07 | 用于基因编辑的修饰的向导rna |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20210087568A1 (https=) |
| EP (1) | EP3802828A4 (https=) |
| JP (2) | JP7832761B2 (https=) |
| KR (2) | KR102919364B1 (https=) |
| CN (1) | CN112567036A (https=) |
| AU (2) | AU2019282824C1 (https=) |
| BR (1) | BR112020024731A2 (https=) |
| CA (1) | CA3102950A1 (https=) |
| CO (1) | CO2021000051A2 (https=) |
| IL (2) | IL278822B1 (https=) |
| MX (2) | MX2020013293A (https=) |
| PH (1) | PH12020552100A1 (https=) |
| SA (1) | SA520420729B1 (https=) |
| SG (1) | SG11202011539VA (https=) |
| TW (1) | TW202016306A (https=) |
| WO (1) | WO2019237069A1 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117925623A (zh) * | 2024-03-20 | 2024-04-26 | 北京引正基因科技有限公司 | 用于hao1基因编辑和治疗ph1的组合物 |
| WO2025007951A1 (zh) * | 2023-07-06 | 2025-01-09 | 上海交通大学 | 一种靶向ttr的sacas9 sgrna及其修饰方式 |
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| IL314915B1 (en) | 2017-01-23 | 2026-02-01 | Regeneron Pharma | Variants of 17-beta-hydroxysteroid dehydrogenase and their uses |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| WO2019028032A1 (en) | 2017-07-31 | 2019-02-07 | Regeneron Pharmaceuticals, Inc. | EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF |
| CN111182790A (zh) | 2017-07-31 | 2020-05-19 | 瑞泽恩制药公司 | Crispr报告体非人类动物及其用途 |
| MX394999B (es) | 2017-09-29 | 2025-03-24 | Regeneron Pharma | Animales no humanos que comprenden un locus ttr humanizado y metodos de uso |
| US10961583B2 (en) | 2017-10-11 | 2021-03-30 | Regeneron Phramaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| CN111885915B (zh) | 2018-03-19 | 2023-04-28 | 瑞泽恩制药公司 | 使用crispr/cas系统对动物进行转录调制 |
| JP7507093B2 (ja) | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 17β-水酸化ステロイド脱水素酵素13型(HSD17B13)iRNA組成物およびその使用方法 |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス,インコーポレーテッド | 第ix因子を発現するための組成物及び方法 |
| EP3946598A1 (en) * | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
| US11891618B2 (en) | 2019-06-04 | 2024-02-06 | Regeneron Pharmaceuticals, Inc. | Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| US11622547B2 (en) | 2019-06-07 | 2023-04-11 | Regeneran Pharmaceuticals, Inc. | Genetically modified mouse that expresses human albumin |
| US11845957B2 (en) | 2019-06-14 | 2023-12-19 | Regeneron Pharmaceuticals, Inc. | Models of tauopathy |
| WO2021026318A2 (en) * | 2019-08-06 | 2021-02-11 | The Penn State Research Foundation | Engineered crispr/cas9 systems for simultaneous long-term regulation of multiple targets |
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| EP4438729A3 (en) * | 2019-12-09 | 2025-03-26 | Caribou Biosciences, Inc. | Crispr abasic restricted nucleotides and crispr accuracy via analogs |
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| JP2023512758A (ja) | 2020-02-07 | 2023-03-29 | インテリア セラピューティクス,インコーポレイテッド | カリクレイン(klkb1)遺伝子編集のための組成物および方法 |
| GB2632565B (en) * | 2020-04-09 | 2025-06-04 | Verve Therapeutics Inc | Base editing of PCSK9 and methods of using same for treatment of disease |
| WO2021231606A2 (en) * | 2020-05-12 | 2021-11-18 | University Of Massachusetts | Modified guide rnas for crispr genome editing |
| CA3204997A1 (en) | 2020-12-11 | 2022-06-16 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class ii in a cell |
| CR20230305A (es) | 2020-12-11 | 2023-11-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación |
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| EP3802828A4 (en) | 2022-10-26 |
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