CN115943209A - 激活γ-珠蛋白基因表达的方法和组合物 - Google Patents

激活γ-珠蛋白基因表达的方法和组合物 Download PDF

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CN115943209A
CN115943209A CN202080032995.3A CN202080032995A CN115943209A CN 115943209 A CN115943209 A CN 115943209A CN 202080032995 A CN202080032995 A CN 202080032995A CN 115943209 A CN115943209 A CN 115943209A
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ssodn
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sequence
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梁峻彬
古博
徐辉
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Guangzhou Ruifeng Biotechnology Co ltd
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Abstract

本发明涉及一种激活γ‑珠蛋白基因转录的新方法。本发明采用含有GATA或其反义互补序列TATC的单链寡核苷酸(ssODN)作为指导信息，在γ‑珠蛋白基因启动子区进行基因编辑形成含有GATA的增强子元件，能够促进γ‑珠蛋白基因在成熟红细胞中表达。利用本发明技术进行基因编辑后的造血干细胞具有正常功能，且在分化成红细胞后能显著提高胎儿血红蛋白的表达，因此可用于β‑地中海贫血和镰刀红细胞贫血的临床治疗。

Description

PCT国内申请，说明书已公开。

Claims (29)

  1. PCT国内申请，权利要求书已公开。
CN202080032995.3A 2020-07-01 2020-07-14 激活γ-珠蛋白基因表达的方法和组合物 Pending CN115943209A (zh)

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WO2022272042A2 (en) * 2021-06-25 2022-12-29 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Primordial germ cells
CN115851706A (zh) * 2021-09-24 2023-03-28 华东师范大学 一种碱基编辑系统及其应用
CN114848851A (zh) * 2022-04-29 2022-08-05 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） 治疗β-地中海贫血的药物
CN116790593B (zh) * 2023-06-28 2024-05-10 贵州医科大学 一种激活γ珠蛋白表达的sgRNA及其CRISPR/Cas9复合体与应用

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MX2018011114A (es) * 2016-03-14 2019-02-20 Editas Medicine Inc Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
AU2018352592A1 (en) * 2017-10-16 2020-06-04 Beam Therapeutics, Inc. Uses of adenosine base editors
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
CN110106203B (zh) * 2019-05-24 2023-08-11 中国医学科学院血液病医院（血液学研究所） 一种新型hbb过表达载体及其设计方法和应用

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