CN111278977A - 一种提高胎儿血红蛋白表达水平的方法 - Google Patents

一种提高胎儿血红蛋白表达水平的方法 Download PDF

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CN111278977A
CN111278977A CN201880069637.2A CN201880069637A CN111278977A CN 111278977 A CN111278977 A CN 111278977A CN 201880069637 A CN201880069637 A CN 201880069637A CN 111278977 A CN111278977 A CN 111278977A
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hematopoietic stem
stem cells
gene editing
cells
sgrna
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CN111278977B (zh
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袁鹏飞
方日国
于玲玲
夏鹏辉
王佳
梁福才
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Guangzhou Ji Yin Medical Technology Co ltd
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Edigene Beijing Biotechnology Co ltd
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Abstract

提供一种基因编辑造血干细胞BCL11A的增强子位点的方法。基因编辑改造后的造血干细胞具有正常细胞功能，能显著提高胎儿血红蛋白的表达，用于β‑地中海贫血和镰刀红细胞贫血的治疗。

Description

PCT国内申请，说明书已公开。

Claims (39)

  1. PCT国内申请，权利要求书已公开。
CN201880069637.2A 2017-10-27 2018-10-26 一种提高胎儿血红蛋白表达水平的方法 Active CN111278977B (zh)

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CN201711116506.9A Active CN109722415B (zh) 2017-10-27 2017-11-13 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
CN201711115779.1A Pending CN109722414A (zh) 2017-10-27 2017-11-13 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
CN201811256120.2A Active CN109735497B (zh) 2017-10-27 2018-10-26 一种提高胎儿血红蛋白表达水平的方法
CN201880069637.2A Active CN111278977B (zh) 2017-10-27 2018-10-26 一种提高胎儿血红蛋白表达水平的方法
CN202210384744.2A Pending CN115747165A (zh) 2017-10-27 2018-10-26 一种提高胎儿血红蛋白表达的方法
CN202210385997.1A Pending CN114921415A (zh) 2017-10-27 2018-10-26 一种提高胎儿血红蛋白表达的方法
CN201880069636.8A Active CN111278976B (zh) 2017-10-27 2018-10-26 一种提高胎儿血红蛋白表达的方法
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