Yoshino H, Hashizume K, Kobayashi E
Division of Organ Replacement Research, Center for Molecular Medicine, Jichi Medical University, Tochigi, Japan.
Gene Ther. 2006 Dec;13(24):1696-702. doi: 10.1038/sj.gt.3302833. Epub 2006 Jul 27.
The naked plasmid DNA transfer method of rapid injection with large volume has been useful for gene therapy in experimental study. However, only small animals like rodents have usually been reported on. In this study, the authors attempted to transfect naked plasmid DNA to the porcine liver by modified hydrodynamic method. We decided to transfer plasmid DNA to a part of the liver using the angio-catheter to reduce the liver damage. To discern the condition of injection, naked plasmid DNA-encoding green fluorescent protein (GFP) was transferred for use as a marker gene. The GFP gene expression was markedly observed in gene-transferred pig livers. In large animals, not only the naked gene quantity, the solution volume containing the plasmid DNA and the injection speed, but also the additional treatments of the portal vein and the hepatic artery preparation were crucial. We found that the following injection condition were needed: plasmid DNA, 3 mg; the solution volume, 150 ml and the injection speed, 5 ml/s. The portal vein and the hepatic artery were clamped during gene delivery and the blood flow of the portal vein was flushed out using normal saline. Cytotoxic T-lymphocyte antigen 4-immunoglobulin (CTLA4-Ig) gene was used to test for secretory protein. CTLA4-Ig gene was injected with a large volume of solution via the hepatic vein to the left outer lobe of the liver selectively. CTLA4-Ig was detected in the pig blood at a maximum serum level of 161.7 ng/ml 1 day after gene transfer, and the CTLA4-Ig was detected for several weeks. Our new technique of inserting a catheter into only a selected portion of the liver reduced liver toxicity and increased gene transfer efficiency. This is the first report of successful gene transfer, using a hydrodynamic method, to the segmental liver in pigs, and achieved more than enough secretory protein for the clinically therapeutic level in pigs.
大容量快速注射裸质粒DNA转移方法在实验研究的基因治疗中很有用。然而,通常报道的仅为啮齿类等小动物。在本研究中,作者尝试通过改良的流体动力学方法将裸质粒DNA转染至猪肝。我们决定使用血管导管将质粒DNA转移至部分肝脏以减少肝损伤。为了辨别注射情况,转移编码绿色荧光蛋白(GFP)的裸质粒DNA用作标记基因。在基因转移的猪肝中明显观察到GFP基因表达。在大型动物中,不仅裸基因量、含质粒DNA的溶液体积和注射速度,而且门静脉和肝动脉准备的额外处理都至关重要。我们发现需要以下注射条件:质粒DNA 3mg;溶液体积150ml,注射速度5ml/s。在基因递送期间夹闭门静脉和肝动脉,并用生理盐水冲洗门静脉血流。使用细胞毒性T淋巴细胞抗原4-免疫球蛋白(CTLA4-Ig)基因检测分泌蛋白。通过肝静脉将大量溶液选择性地注射CTLA4-Ig基因至肝左外叶。基因转移后1天,猪血液中检测到CTLA4-Ig,血清最高水平为161.7ng/ml,并且CTLA4-Ig在数周内均可检测到。我们仅将导管插入肝脏选定部分的新技术降低了肝毒性并提高了基因转移效率。这是首次使用流体动力学方法成功将基因转移至猪的节段性肝脏的报道,并且获得了超过猪临床治疗水平所需的足够分泌蛋白。