Huard J, Lochmüller H, Acsadi G, Jani A, Massie B, Karpati G
Montreal Neurological Institute, Quebec, Canada.
Gene Ther. 1995 Mar;2(2):107-15.
One of the key factors that determines the efficacy of adenovirus-mediated gene therapy in genetic diseases, is the degree and extent of transduction of the target cells by adenovirus (AV)-recombinants carrying the therapeutic gene or cDNA. In this paper we provide experimental evidence which indicates that the route of administration of the AV-recombinants has a major influence on the transduction of various tissues in young rats. The heart, diaphragm, intercostal muscles and thymus show high transduction after intra-arterial (left cardiac ventricle) injection. By contrast, the liver shows a high transduction after intravenous injection. A substantial viremia develops within 2 h of gastric-rectal, intraperitoneal and intracardiac administration of AV recombinants. The number of adenoviral DNA copies per nucleus of transduced cells ranged from one to three in most tissues. These numbers correlated well with the overall transduction efficiency of the tissue determined by reporter gene expression. The various factors that determine which route of administration favors a high transduction rate in a particular tissue can be analyzed and this can lead to an improved efficiency of gene therapy in targeting a particular tissue in a disease.
决定腺病毒介导的基因疗法对遗传疾病疗效的关键因素之一,是携带治疗性基因或cDNA的腺病毒(AV)重组体对靶细胞的转导程度和范围。在本文中,我们提供了实验证据,表明AV重组体的给药途径对幼鼠各种组织的转导有重大影响。经动脉(左心室)注射后,心脏、膈肌、肋间肌和胸腺显示出高转导率。相比之下,静脉注射后肝脏显示出高转导率。经胃直肠、腹腔和心内注射AV重组体后2小时内会出现大量病毒血症。在大多数组织中,转导细胞每个细胞核中的腺病毒DNA拷贝数为1至3个。这些数字与通过报告基因表达确定的组织总体转导效率密切相关。可以分析决定哪种给药途径有利于特定组织高转导率的各种因素,这可能会提高针对疾病中特定组织的基因治疗效率。