Chen Cang, Guderyon Michael J, Ge Guo, Clark Robert A, Li Senlin
Department of Medicine, UT Health Science Center at San Antonio, San Antonio, TX, USA.
Research and Development Service, Audie L. Murphy VA Hospital, San Antonio, TX, USA.
Methods Mol Biol. 2019;1919:205-213. doi: 10.1007/978-1-4939-9007-8_16.
Lentiviral vectors are increasingly used as efficient gene transfer tools in the experimental and clinical gene therapy treatment of acquired and inherited genetic diseases. Hematopoietic stem cells (HSCs) are characterized by the capacity for self-renewal, as well as multi-lineage differentiation and maintenance of the lymphohematopoietic system throughout life. As such, HSC transplantation (HSCT) has proven to be a powerful therapeutic modality for the treatment of both malignant and nonmalignant disorders. Transduction of lentiviral vectors into HSCs may offer long-term stable expression of a therapeutic gene in both preclinical and clinical settings. The purpose of this chapter is to describe an optimized procedure for lentiviral transduction of mouse HSCs followed by HSCT.
慢病毒载体作为一种高效的基因转移工具，在获得性和遗传性疾病的实验性和临床基因治疗中越来越多地被使用。造血干细胞（HSCs）的特点是具有自我更新能力，以及在整个生命过程中多谱系分化和维持淋巴造血系统。因此，造血干细胞移植（HSCT）已被证明是治疗恶性和非恶性疾病的一种强有力的治疗方式。将慢病毒载体转导到造血干细胞中，在临床前和临床环境中都可能提供治疗基因的长期稳定表达。本章的目的是描述一种优化的程序，用于小鼠造血干细胞的慢病毒转导，随后进行造血干细胞移植。
