Gene therapy for neuroblastoma by using Gene Gun. Gene transfer in the spleen.

背景/目的：宫内基因治疗比出生后基因治疗具有许多潜在的优势。一种在子宫内将基因转移到胎儿的新方法使用了一种称为基因枪的新工具。基因枪比其他子宫内方法侵入性更小，更简单。本研究的目的是确定基因枪是否是一个有效的工具，用于转移基因到小鼠胎儿在utero.Methods：使用基因枪，我们转移质粒，其中包括增强型绿色荧光蛋白（EGFP）基因和巨细胞病毒启动子的腹部皮肤的40 A/J胎鼠在每个三个胎龄（13，14，或15天）。基因转移后4或5天，计数存活胎儿的数量，并通过荧光显微镜分析皮肤中EGFP的彩色图像以获得基因转移率。结果：转基因组平均存活率为89.2%（107/120），对照组平均存活率为91.7%（55/60）。基因转移率在胎龄14天时最高，为100%（37/37）。结论：基因枪宫内基因转移技术是一种创伤小的基因转移技术，是一种有效的宫内基因转移工具

Background/Purpose: In utero gene therapy offers a number of potential advantages over postnatal gene therapy. A new method of gene transfer to fetuses in utero uses a new tool called a gene gun. The gene gun is less invasive and simpler than other in utero methods. The present study was designed to determine whether the gene gun is an effective tool for transferring genes to mouse fetuses in utero.Methods: Using a gene gun, we transferred plasmids that included enhanced green fluorescent protein (EGFP) genes and cytomegalo virus promoters to the abdominal skin of 40 A/J fetal mice at each of three gestational ages (13, 14, or 15 days). Four or 5 days after gene transfer, the number of surviving fetuses was counted and a color image of EGFP in the skin was analyzed for gene transfer rates by fluorescence microscopy. Survival rates were analyzed using Fisher s exact test.Results: The mean survival rate was 89.2% (107/120) in gene transfer fetuses and 91.7% (55/60) in controls. The highest gene transfer rate was 100% (37/37) at the gestational age of 14 days. The rate was 97.1% (34/35) at gestational ages of 13 and 15 days.Conclusion: This study shows that in utero gene transfer by gene gun is less invasive technique and the gene gun is an effective tool transferring genes to mouse fetuses in utero