摘要
造血干细胞(hematopoietic stem cells, HSCs)是一类多能干细胞,位于特殊的造血微环境,主要存在于骨髓中。其能自我更新和多向分化为各种功能的血细胞,维持血液系统的建立和动态平衡。造血干细胞的这些重要特性以及造血干细胞移植在临床上的广泛应用,结合基因治疗和基因编辑技术的进步,使得基于造血干细胞治疗多种血液疾病和免疫疾病的基因治疗研究在近年来取得了很大的进展。该文将从造血干细胞生物学特征、来源、造血干细胞微环境的基础研究,以及造血干细胞基因治疗、自体造血干细胞移植治疗β-地中海贫血等方面的临床研究和应用进展进行综述。
Multipotent hematopoietic stem cells(HSCs)reside in specialized niche mainly in adult bone marrow with extensive self-renewal and differentiation capacity,can replenish all myeloid and lymphoid blood cell lineages and maintain blood system establishment and dynamic balance.Due to these important characteristics of HSCs and extensive clinical application of HSC transplantation,combined with advances in gene therapy and gene editing,the research of HSCs based gene therapy for many blood diseases and immune diseases has made great progress in recent years.In this review,we focus on self-renewal and differentiation of HSCs,sources and microenvironment of HSCs,gene editing based clinical research with HSCs especially for beta-Thalassemia.
作者
田晓玲
杨菲
吴宇轩
Tian Xiaoling;Yang Fei;Wu Yuxuan(Shanghai Key Laboratory of Regulatory Biology,Institute of Biomedical Sciences and School of Life Sciences,East China Normal University,Shanghai 200241,China)
出处
《中国细胞生物学学报》
CAS
CSCD
2019年第6期1003-1011,共9页
Chinese Journal of Cell Biology
关键词
造血干细胞
自我更新
分化
基因编辑
基因治疗
地中海贫血
hematopoietic stem cells
self-renewal
differentiation
gene editing
gene therapy
Thalassemia
