CN114364799A - 编辑造血干/祖细胞中bcl11a基因的方法 - Google Patents

编辑造血干/祖细胞中bcl11a基因的方法 Download PDF

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CN114364799A
CN114364799A CN202080058741.9A CN202080058741A CN114364799A CN 114364799 A CN114364799 A CN 114364799A CN 202080058741 A CN202080058741 A CN 202080058741A CN 114364799 A CN114364799 A CN 114364799A
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seq
cells
grna
edited
hematopoietic stem
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焦娇
崔正之
栗飞红
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Gan and Lee Pharmaceuticals Co Ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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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  • Health & Medical Sciences (AREA)
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Abstract

本发明提供了编辑造血干/祖细胞中BCL11A基因的方法及提高人造血干/祖细胞经红系分化后HbF表达的方法，本发明还提供了BCL11A被编辑的造血干/祖细胞及所述细胞用于制备预防或治疗贫血性疾病的药物或医用制品中的用途，还提供了用于编辑造血干/祖细胞BCL11A的gRNA及包含所述gRNA的试剂盒。

Description

PCT国内申请，说明书已公开。

Claims (20)

  1. PCT国内申请，权利要求书已公开。
CN202080058741.9A 2019-08-28 2020-08-28 编辑造血干/祖细胞中bcl11a基因的方法 Pending CN114364799A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2019107990486 2019-08-28
CN201910799048 2019-08-28
PCT/CN2020/112224 WO2021037232A1 (zh) 2019-08-28 2020-08-28 编辑造血干/祖细胞中bcl11a基因的方法

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CN114364799A true CN114364799A (zh) 2022-04-15

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WO (1) WO2021037232A1 (zh)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023107675A2 (en) * 2021-12-10 2023-06-15 The Children's Medical Center Corporation Combination bcl11a enhancer editing

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108779462A (zh) * 2015-12-28 2018-11-09 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3445388B1 (en) * 2016-04-18 2024-04-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN109706148A (zh) * 2017-09-30 2019-05-03 广东赤萌医疗科技有限公司 一种用于敲除BCL11A基因或者BCL11A基因增强子的gRNA、gRNA组合物以及电转方法
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108779462A (zh) * 2015-12-28 2018-11-09 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MATTHEW C. CANVER等: "BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis" *
高飏: "CRISPR_Cas9编辑CD34_造血干细胞BCL11A基因提升HbF表达" *

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