CN114269941A - 基于脱靶评估评价基因编辑治疗的方法 - Google Patents

基于脱靶评估评价基因编辑治疗的方法 Download PDF

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CN114269941A
CN114269941A CN202080058282.4A CN202080058282A CN114269941A CN 114269941 A CN114269941 A CN 114269941A CN 202080058282 A CN202080058282 A CN 202080058282A CN 114269941 A CN114269941 A CN 114269941A
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target marker
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袁鹏飞
方日国
金鸣
张永建
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Guangzhou Ji Yin Medical Technology Co ltd
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Abstract

一种确定细胞疗法对个体的适合性的方法。利用生物信息学方法和无偏向的潜在脱靶位点实验分析方法,在向个体施用细胞疗法前对源自个体的基因修饰细胞的潜在脱靶位点进行预测,并在将源自个体的基因修饰细胞施用至个体后对潜在脱靶位点进行监测,确定所述潜在脱靶位点否发生基因编辑,并基于基因编辑事件的发生评估所述治疗的适合性。

Description

PCT国内申请,说明书已公开。

Claims (35)

  1. PCT国内申请,权利要求书已公开。
CN202080058282.4A 2019-09-04 2020-09-04 基于脱靶评估评价基因编辑治疗的方法 Pending CN114269941A (zh)

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CNPCT/CN2019/104303 2019-09-04
CN2019104303 2019-09-04
PCT/CN2020/113562 WO2021043278A1 (zh) 2019-09-04 2020-09-04 基于脱靶评估评价基因编辑治疗的方法

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US20220307020A1 (en) 2019-04-15 2022-09-29 Edigene Inc. Methods and compositions for editing rnas
EP3997229A4 (en) 2019-07-12 2024-07-03 Univ Beijing TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109735574A (zh) * 2017-10-27 2019-05-10 博雅辑因(北京)生物科技有限公司 一种提高胎儿血红蛋白表达的方法
CN109789185A (zh) * 2016-07-28 2019-05-21 基础科学研究院 用于治疗眼部疾病的含cas9蛋白和向导rna的药物组合物

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE038850T2 (hu) 2012-05-25 2018-11-28 Univ California Eljárások és kompozíciók cél-DNS RNS-irányított módosításához és transzkripció RNS-irányított modulálásához
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
CN110066775B (zh) 2012-10-23 2024-03-19 基因工具股份有限公司 用于切割靶dna的组合物及其用途
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN108351350B (zh) * 2015-08-25 2022-02-18 杜克大学 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法
EP3450570B1 (en) * 2016-04-28 2021-03-10 Industry-Academic Cooperation Foundation, Yonsei University Method for evaluating, in vivo, activity of rna-guided nuclease in high-throughput manner
MX2018013445A (es) * 2016-05-06 2019-09-09 Juno Therapeutics Inc Celulas diseñadas geneticamente y metodos para obtener las mismas.
KR102558931B1 (ko) * 2017-06-23 2023-07-21 인스크립타 인코포레이티드 핵산 가이드 뉴클레아제
WO2019052577A1 (zh) * 2017-09-18 2019-03-21 博雅辑因(北京)生物科技有限公司 一种基因编辑t细胞及其用途
EP3697907A1 (en) * 2017-10-17 2020-08-26 CRISPR Therapeutics AG Compositions and methods for gene editing for hemophilia a
CN107967411B (zh) * 2017-11-21 2021-09-10 南方科技大学 一种脱靶位点的检测方法、装置及终端设备
CN109868283B (zh) * 2019-02-21 2021-07-20 浙江农林大学 一种评估CRISPR/Cas9基因编辑效率或脱靶频率的方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109789185A (zh) * 2016-07-28 2019-05-21 基础科学研究院 用于治疗眼部疾病的含cas9蛋白和向导rna的药物组合物
CN109735574A (zh) * 2017-10-27 2019-05-10 博雅辑因(北京)生物科技有限公司 一种提高胎儿血红蛋白表达的方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
王影等: "CRISPR/Cas9基因组定点编辑中脱靶现象的研究进展", 植物学报, vol. 53, no. 4, pages 528 - 541 *

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US20220340969A1 (en) 2022-10-27
KR20220053671A (ko) 2022-04-29
WO2021043278A1 (zh) 2021-03-11
EP4026910A1 (en) 2022-07-13
JP2022547105A (ja) 2022-11-10
CA3150230A1 (en) 2021-03-11
AU2020342207A1 (en) 2022-04-21

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