Development of methods that allow an efficient expression of exogenous genes in animals would provide tools for gene function studies, treatment of diseases and for obtaining gene products. Therefore, we have developed a hydrodynamics-based procedure for expressing transgenes in mice by systemic administration of plasmid DNA. Using cDNA of luciferase and beta-galactosidase as a reporter gene, we demonstrated that an efficient gene transfer and expression can be achieved by a rapid injection of a large volume of DNA solution into animals via the tail vein. Among the organs expressing the transgene, the liver showed the highest level of gene expression. As high as 45 microg of luciferase protein per gram of liver can be achi- eved by a single tail vein injection of 5 microg of plasmid DNA into a mouse. Histochemical analysis using beta-galactosidase gene as a reporter reveals that approximately 40percent of hepatocytes express the transgene. The time-response curve shows that the level of transgene expression in the liver reaches the peak level in approximately 8 h after injection and decreases thereafter. The peak level of gene expression can be regained by repeated injection of plasmid DNA. These results suggest that a simple, convenient and efficient method has been developed and which can be used as an effective means for studying gene function, gene regulation and molecular pathophysiology through gene transfer, as well as for expressing proteins in animals.

F Liu

Y Song

D Liu

Gene therapy

基因疗法涵盖了基于遗传成分的新型治疗技术的研究和临床应用。在过去的几十年里，从识别导致疾病的新遗传靶点到显示治疗益处的临床研究，技术的重大进步已将这一多学科领域提升到现代医学的前沿。

Gene Therapy is a field that focuses on the research and clinical application of novel therapeutic techniques based on genetic components. In recent decades, significant advancements in technology have elevated this multidisciplinary field to the forefront of modern medicine, from identifying new genetic targets that cause diseases to demonstrating therapeutic benefits in clinical studies.

基因疗法是研究和临床应用基于遗传成分的新型治疗技术的领域。近年来，随着对导致疾病的新遗传靶点的识别以及临床研究中治疗效果的展示，这一多学科领域的技术进步显著，已将其提升至现代医学的前沿。

Gene Therapy

Gene Ther

Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA.

A hydrodynamics-based procedure for expressing transgenes in mice by systemic administration of plasmid DNA is developed and which can be used as an effective means for studying gene function, gene regulation and molecular pathophysiology through gene transfer, as well as for expressing proteins in animals.

Category	Quartile
医学	3
医学, 生物工程与应用微生物	2
医学, 生化与分子生物学	3
医学, 遗传学	3
医学, 医学研究与实验	3

Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA.

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Journal Info

Category	Quartile
MEDICINE, RESEARCH & EXPERIMENTAL	2