Johns D C, Nuss H B, Chiamvimonvat N, Ramza B M, Marban E, Lawrence J H
Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA.
J Clin Invest. 1995 Aug;96(2):1152-8. doi: 10.1172/JCI118103.
Excitability is governed primarily by the complement of ion channels in the cell membrane that shape the contour of the action potential. To modify excitability by gene transfer, we created a recombinant adenovirus designed to overexpress a Drosophila Shaker potassium channel (AdShK). In vitro, a variety of mammalian cell types infected with AdShK demonstrated robust expression of the exogenous channel. Spontaneous action potentials recorded from cardiac myocytes in primary culture were abbreviated compared with noninfected myocytes. Intravascular infusion of AdShK in neonatal rats induced Shaker potassium channel mRNA expression in the liver, and large potassium currents could be recorded from explanted hepatocytes. Thus, recombinant adenovirus technology has been used for in vitro and in vivo gene transfer of ion channel genes designed to modify cellular action potentials. With appropriate targeting, such a strategy may be useful in gene therapy of arrhythmias, seizure disorders, and myotonic muscle diseases.
兴奋性主要由细胞膜中形成动作电位轮廓的离子通道的互补性所决定。为了通过基因转移来改变兴奋性,我们构建了一种重组腺病毒,旨在过表达果蝇Shaker钾通道(AdShK)。在体外,感染AdShK的多种哺乳动物细胞类型都表现出外源通道的强劲表达。与未感染的心肌细胞相比,原代培养的心肌细胞记录到的自发动作电位缩短。新生大鼠血管内注入AdShK可诱导肝脏中Shaker钾通道mRNA表达,并且从分离出的肝细胞中可记录到较大的钾电流。因此,重组腺病毒技术已被用于离子通道基因的体外和体内基因转移,这些基因旨在改变细胞动作电位。通过适当的靶向作用,这种策略可能在心律失常、癫痫症和强直性肌病的基因治疗中有用。