摘要
造血干细胞具有自我更新和分化为各类血细胞的潜能,一直被认为是最理想的基因治疗靶细胞之一.近年来,基于慢病毒载体的造血干细胞基因治疗逐渐进入临床.同时,随着CRISPR-Cas9等基因编辑技术的不断发展,第二代造血干细胞精准基因治疗研究已经取得重要进展,预计将逐渐开始临床转化.但目前,造血干细胞基因编辑还面临一些问题亟待解决,尤其是精准编辑效率还需要大幅度提高.
Hematopoietic stem cells possess the capacity of self-renewal and differentiation into all lineages of blood cells and have been recognized as one of the most ideal cells for gene therapy. Over the last decade, significant progress has been made in lentiviral vector-based hematopoietic stem cell gene therapy, and long-term success has been achieved in some clinical trials. The recent advent of the CRISPR-Cas9 genome editing technology has galvanized the field of gene therapy, and emerging on the horizon is targeted gene therapy by precise genome editing. However, to fulfill the vision of the second-generation hematopoietic stem cell gene therapy, further innovation is imperative to considerably increase the efficiency of precision genome editing in long-term hematopoietic stem cells.
作者
张健萍
程涛
张孝兵
ZHANG JianPing;CHENG Tao;ZHANG Xiao-Bing(State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China;Center for Stem Cell Medicine, Chinese Academy of Medical Sciences, Tianjin 300020, China;Department of Stem Cell ＆ Regenerative Medicine, Peking Union Medical College, Tianjin 300020, China;Collaborative Innovation Center for Cancer Medicine, Tianjin 300020, China;Department of Medicine, Loma Linda University, Loma Linda, CA 92350, USA)
出处
《中国科学：生命科学》
CSCD
北大核心
2017年第12期1323-1335,共13页
Scientia Sinica(Vitae)
基金
国家重点研发计划(批准号:2016YFA0100600)
科技部重大基础研究项目(批准号:2015CB964902)
国家自然科学基金(批准号:81500148,81400150)
中国医学科学院医学与健康科技创新工程服务“一带一路”战略先导科研专项(批准号:2017-I2M-B&R-04)
中国医学科学院中央级公益性科研院所基本科研业务费(批准号:2016ZX310184-3)资助
