Hendrie Paul C, Russell David W
Division of Hematology, Department of Medicine, University of Washington, Seattle, WA 98195, USA.
Mol Ther. 2005 Jul;12(1):9-17. doi: 10.1016/j.ymthe.2005.04.006.
Genetic manipulation of cells for scientific and therapeutic goals can be achieved by both gene-addition and gene-targeting methods. Gene targeting precisely alters a gene in its natural chromosome location, providing distinct advantages over gene-addition approaches. Classic gene-targeting delivery systems (microinjection, electroporation, or calcium phosphate transfection) have led to major scientific advances, but are too inefficient in their current state to be used for some applications, including gene therapy. This review describes the development of gene-targeting vectors based on three types of viruses (retrovirus, adenovirus, and adeno-associated virus) and discusses the design, possible mechanisms of action, and applications of gene-targeting vectors based on adeno-associated virus.
为实现科学和治疗目标而对细胞进行基因操作,可通过基因添加和基因靶向方法来完成。基因靶向能精确改变基因在其自然染色体位置上的状态,与基因添加方法相比具有明显优势。经典的基因靶向递送系统(显微注射、电穿孔或磷酸钙转染)已带来了重大的科学进展,但就目前状况而言,其效率过低,无法用于包括基因治疗在内的某些应用。本综述描述了基于三种病毒(逆转录病毒、腺病毒和腺相关病毒)的基因靶向载体的发展,并讨论了基于腺相关病毒的基因靶向载体的设计、可能的作用机制及应用。