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1. CN111876416 - Method and composition for activating gamma-globin gene expression

Office
China
Application Number 202010616648.7
Application Date 01.07.2020
Publication Number 111876416
Publication Date 03.11.2020
Grant Number 111876416
Grant Date 03.09.2021
Publication Kind B
IPC
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KPEPTIDES
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0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
Applicants GUANGZHOU REFORGENE BIOLOGICAL TECHNOLOGY CO., LTD.
广州瑞风生物科技有限公司
Inventors LIANG JUNBIN
梁峻彬
GU BO
古博
XU HUI
徐辉
Agents 广州三环专利商标代理有限公司 44202
广州三环专利商标代理有限公司 44202
Title
(EN) Method and composition for activating gamma-globin gene expression
(ZH) 激活γ-珠蛋白基因表达的方法和组合物
Abstract
(EN) The invention relates to a novel method for activating gamma globin gene transcription. Single-stranded oligonucleotide (ssODN) containing GATA or an antisense complementary sequence TATC thereof is used as guidance information, gene editing is performed in a gamma globin gene regulation region to form an enhancer element containing GATA, and expression of a gamma globin gene in mature red blood cells can be promoted. Hematopoietic stem cells obtained after gene editing through the technology have normal functions, expression of fetal hemoglobin can be remarkably improved after the hematopoietic stem cells are differentiated into erythrocytes, and therefore the hematopoietic stem cells can be used for clinical treatment of beta thalassemia and sickle erythrocyte anemia.
(ZH) 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‑珠蛋白基因调控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。
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