Lukashev A N, Zamyatnin A A
Chumakov Institute of Poliomyelitis and Viral Encephalitides, Moscow, 142782, Russia.
Biochemistry (Mosc). 2016 Jul;81(7):700-8. doi: 10.1134/S0006297916070063.
Gene therapy is the straightforward approach for the application of recent advances in molecular biology into clinical practice. One of the major obstacles in the development of gene therapy is the delivery of the effector to and into the target cell. Unfortunately, most methods commonly used in laboratory practice are poorly suited for clinical use. Viral vectors are one of the most promising methods for gene therapy delivery. Millions of years of evolution of viruses have resulted in the development of various molecular mechanisms for entry into cells, long-term survival within cells, and activation, inhibition, or modification of the host defense mechanisms at all levels. The relatively simple organization of viruses, small genome size, and evolutionary plasticity allow modifying them to create effective instruments for gene therapy approaches. This review summarizes the latest trends in the development of gene therapy, in particular, various aspects and prospects of the development of clinical products based on viral delivery systems.
基因治疗是将分子生物学的最新进展应用于临床实践的直接方法。基因治疗发展的主要障碍之一是将效应物递送至靶细胞并进入靶细胞。不幸的是,实验室实践中常用的大多数方法都不太适合临床应用。病毒载体是基因治疗递送中最有前景的方法之一。数百万年的病毒进化导致了各种进入细胞、在细胞内长期存活以及在各个层面激活、抑制或修饰宿主防御机制的分子机制的发展。病毒相对简单的结构、较小的基因组大小和进化可塑性使得对其进行改造以创建用于基因治疗方法的有效工具成为可能。本综述总结了基因治疗发展的最新趋势,特别是基于病毒递送系统的临床产品开发的各个方面和前景。