Athanasopoulos Takis, Munye Mustafa M, Yáñez-Muñoz Rafael J
Cell and Gene Therapy Discovery Research, Platform Technology and Sciences, GSK Medicines Research Centre, Gunnels Wood Road, Stevenage, Hertfordshire SG1 2NY, UK.
AGCTlab.org, Centre for Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London, Egham, Surrey TW20 0EX, UK.
Hematol Oncol Clin North Am. 2017 Oct;31(5):753-770. doi: 10.1016/j.hoc.2017.06.007.
Gene delivery vectors that do not rely on host cell genome integration offer several advantages for gene transfer, chiefly the avoidance of insertional mutagenesis and position effect variegation. However, unless engineered for replication and segregation, nonintegrating vectors will dilute progressively in proliferating cells, and are not exempt of epigenetic effects. This article provides an overview of the main nonintegrating viral (adenoviral, adeno-associated viral, integration-deficient retro-lentiviral, poxviral), and nonviral (plasmid vectors, artificial chromosomes) vectors used for preclinical and clinical cell and gene therapy applications. Particular emphasis is placed on their use in hematologic disease.
不依赖宿主细胞基因组整合的基因传递载体在基因转移方面具有多个优势，主要包括避免插入诱变和位置效应斑驳现象。然而，除非经过工程改造以实现复制和分离，否则非整合型载体在增殖细胞中会逐渐稀释，并且无法免除表观遗传效应。本文概述了用于临床前和临床细胞及基因治疗应用的主要非整合型病毒载体（腺病毒、腺相关病毒、整合缺陷型逆转录慢病毒、痘病毒）和非病毒载体（质粒载体、人工染色体）。特别强调了它们在血液系统疾病中的应用。
