Romano Gaetano
Department of Neurosurgery, Farber Institute for the Neurosciences, Jefferson Medical College, Thomas Jefferson University, Jefferson Hospital for the Neurosciences, Suite 400, 900 Walnut Street, Philadelphia, PA 19107, USA.
Drug News Perspect. 2005 Mar;18(2):128-34. doi: 10.1358/dnp.2005.18.2.886481.
Lentiviral-based vectors have been widely used lately in preclinical studies and for the in vitro genetic manipulation of embryonic and adult stem cells. They allow for the transduction of nondividing cells and for stable gene expression. On these grounds, lentiviral vectors look promising for eventual applications for the gene-based treatment of neurological disorders, cardiopathies and inherited or acquired genetic diseases. The best developed and characterized lentiviral vector system is based on the human immunodeficiency virus type 1 (HIV-1). However, safety concerns preclude any possible application of HIV-1-derived vectors in clinical trials. For this reason, gene therapists are currently improving the design of other lentiviral vectors, such as feline immunodeficiency virus, equine anemia infectious virus and Visna virus. These lentiviruses are not pathogenic in humans and are only distantly related to primate Retroviridae. This review summarizes the achievements in improving the design of lentiviral vector systems that are not based on HIV-1.
基于慢病毒的载体最近已广泛应用于临床前研究以及胚胎干细胞和成体干细胞的体外基因操作。它们能够转导非分裂细胞并实现稳定的基因表达。基于这些原因,慢病毒载体在神经疾病、心脏病以及遗传性或获得性基因疾病的基因治疗最终应用方面看起来很有前景。目前开发和表征得最好的慢病毒载体系统是基于1型人类免疫缺陷病毒(HIV-1)。然而,安全问题使得HIV-1衍生载体无法在临床试验中得到任何应用。因此,基因治疗专家目前正在改进其他慢病毒载体的设计,如猫免疫缺陷病毒、马传染性贫血病毒和维斯纳病毒。这些慢病毒对人类无致病性,且与灵长类逆转录病毒科只有远缘关系。本综述总结了在改进非基于HIV-1的慢病毒载体系统设计方面所取得的成果。