阅读说明：本技术 一种血红蛋白病治疗有效性预测方法 (Method for predicting treatment effectiveness of hemoglobinopathy ) 是由 方日国 于玲玲 杨卉慧 于 2020-04-29 设计创作，主要内容包括：涉及一种治疗个体中的血红蛋白病的方法,包括：a)评估步骤,该评估步骤包括评估经修饰的CD34阳性造血干细胞/祖细胞的第一群体分化后产生所需水平的γ-球蛋白或胎儿血红蛋白的能力,其中第一群体的经修饰的CD34阳性HSPC来源于所述个体并经过修饰以降低BCL11A功能；并且b)治疗步骤,该治疗步骤包括向所述个体施用第二群体经修饰的CD34阳性HSPC,该经修饰的CD34阳性HSPC来源于所述个体并且经修饰降低了BCL11A功能。同时,还涉及治疗个体中的血红蛋白病的方法、选择患有血红蛋白病的个体用于以第二群体的经修饰CD34阳性HSPC进行治疗的方法和确定患有血红蛋白病的个体是否适合或不适合用来源于所述个体并经修饰降低了BCL11A功能的第二群体的经修饰CD34阳性HSPC进行治疗的方法。(Relates to a method of treating hemoglobinopathy in a subject comprising: a) an evaluation step comprising evaluating a first population of modified CD34 positive hematopoietic stem/progenitor cells for their ability to differentiate to produce a desired level of gamma-globulin or fetal hemoglobin, wherein the modified CD34 positive HSPCs of the first population are derived from the individual and modified to reduce BCL11A function; and b) a treatment step comprising administering to the individual a second population of modified CD34 positive HSPCs derived from the individual and modified to reduce BCL11A function. Also, methods of treating hemoglobinopathies in a subject, methods of selecting subjects having hemoglobinopathies for treatment with a second population of modified CD34 positive HSPCs, and methods of determining whether subjects having hemoglobinopathies are suitable or unsuitable for treatment with a second population of modified CD34 positive HSPCs derived from the subject and modified to reduce BCL11A function are also contemplated.)