基因治疗用质粒DNA生产面临的问题及研究进展  

Research Progress in Large-scale Production of Plasmid DNA for Gene Therapy

作　　者：胡春生[1,2] 张庆林[1] 张通[2] 

机构地区：[1]军事医学科学院放射与辐射医学研究所,北京100850 [2]内蒙古工业大学化工学院,呼和浩特010051

出　　处：《中国生物工程杂志》2011年第4期119-123,共5页China Biotechnology

基　　金：国家"863"计划资助项目(2001AA217061)

摘　　要：基因治疗已成为21世纪一些重大疾病的有效治疗策略,目前携带治疗基因的重组质粒已作为基因药物进入临床研究。对用于基因治疗的生物制品的生产与质量控制都有相当严格的要求。虽然已建立大规模符合药学规格的质粒DNA生产工艺,能满足临床需求,但在这些生产工艺中还存在一些难以克服的瓶颈,如:载体构建、细胞裂解、细菌染色体DNA去除、细菌内毒素去除、生产过程中质量控制等。就近年来大规模生产临床用质粒DNA遇到的相关问题及解决方案作一综述。Plasmid DNA encoding treatment gene for pharmaceutical applications was approved into the clinical trials.A pre-requisite to the success of plasmid-based therapies is the development of effective production of plasmid DNA.However,at present,there are several problems and bottlenecks in large-scale production of pharmaceutical plasmid DNA.Such as vector construction,cell lysis,bacteria chromosome DNA removing,bacteria endotoxin removing and quality-control in production process.The downstream for the large-scale production of plasmid DNA,limitations and the strategies used to obtain a final product that meets specifications was reviewed.

关 键 词：基因治疗 质粒DNA 大规模 碱裂解 纯化 

分 类 号：Q819[生物学—生物工程]