CA3112011A1 - Compositions and methods for delivering a nucleobase editing system - Google Patents
Compositions and methods for delivering a nucleobase editing system Download PDFInfo
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- CA3112011A1 CA3112011A1 CA3112011A CA3112011A CA3112011A1 CA 3112011 A1 CA3112011 A1 CA 3112011A1 CA 3112011 A CA3112011 A CA 3112011A CA 3112011 A CA3112011 A CA 3112011A CA 3112011 A1 CA3112011 A1 CA 3112011A1
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- cas9
- terminal fragment
- fragment
- deaminase
- fused
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| WO2020252455A1 (en) | 2019-06-13 | 2020-12-17 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
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| GB2632564B (en) * | 2020-04-09 | 2025-06-18 | Verve Therapeutics Inc | Base editing of angptl3 and methods of using same for treatment of disease |
| CN116670154A (zh) | 2020-07-24 | 2023-08-29 | 总医院公司 | 增强的病毒样颗粒及使用其递送至细胞的方法 |
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| WO2024233941A1 (en) * | 2023-05-11 | 2024-11-14 | Massachusetts Eye And Ear Infirmary | Base editing approaches to treat abca4-associated stargardt disease |
| WO2024249954A1 (en) | 2023-05-31 | 2024-12-05 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025038955A1 (en) * | 2023-08-16 | 2025-02-20 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Systems and methods for improving safety of split intein aav mediated gene therapy |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
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| US10077453B2 (en) * | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
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| CN106011104B (zh) | 2015-05-21 | 2019-09-27 | 清华大学 | 利用拆分Cas系统进行基因编辑和表达调控方法 |
| WO2016205759A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| US20200340012A1 (en) | 2016-08-18 | 2020-10-29 | The Regents Of The University Of California | Crispr-cas genome engineering via a modular aav delivery system |
| EP3526320A1 (en) * | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| AU2019336245A1 (en) * | 2018-09-07 | 2021-04-01 | Beam Therapeutics Inc. | Compositions and methods for delivering a nucleobase editing system |
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