Barzon Luisa, Stefani Anna-Lisa, Pacenti Monia, Palù Giorgio
Department of Histology, Microbiology and Medical Biotechnologies, University of Padova, Via Gabelli 63, I-35121 Padova, Italy.
Expert Opin Biol Ther. 2005 May;5(5):639-62. doi: 10.1517/14712598.5.5.639.
Several viruses have been engineered for gene therapy applications, and the specific properties of each viral vector have been exploited to target a variety of inherited and acquired diseases. Preclinical and clinical studies demonstrated that viral vectors are highly versatile tools capable of efficient transfer of foreign genetic information into almost all cell types and tissues. Gene therapy applications depend on vector characteristics, such as host range, cell- or tissue-specific targeting, genome integration, efficiency and duration of transgene expression, packaging capacity, and suitability for scale-up production. This review discusses the advances in the development of viral vectors, with particular emphasis on how knowledge of virus biology has been exploited to design a variety of vectors with improved safety characteristics and efficiency, potentially suitable for a large number of gene therapy applications.
几种病毒已被改造用于基因治疗应用,并且每种病毒载体的特定特性已被用于针对多种遗传性和后天性疾病。临床前和临床研究表明,病毒载体是高度通用的工具,能够将外源遗传信息有效转移到几乎所有细胞类型和组织中。基因治疗应用取决于载体特性,如宿主范围、细胞或组织特异性靶向、基因组整合、转基因表达的效率和持续时间、包装能力以及扩大生产的适用性。本综述讨论了病毒载体开发方面的进展,特别强调了如何利用病毒生物学知识来设计具有改进的安全特性和效率的各种载体,这些载体可能适用于大量基因治疗应用。