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

背景/目的：与产后基因治疗相比，子宫内基因治疗具有许多潜在的优势。一种将基因转移到子宫内胎儿的新方法使用了一种叫做基因枪的新工具。基因枪比其他子宫内方法侵入性小，操作简单。本研究旨在确定基因枪是否是将基因转移到子宫内小鼠胎儿的有效工具。方法：利用基因枪将含有增强型绿色荧光蛋白（EGFP）基因和巨细胞病毒启动子的质粒转移到3个胎龄（13、14和15天）的40只a /J胎鼠的腹部皮肤上。基因转移后4天或5天，计数存活胎数，并通过荧光显微镜分析皮肤中EGFP的彩色图像，观察基因转移率。生存率采用Fisher精确检验。结果：基因移植胎的平均存活率为89.2%(107/120)，对照组为91.7%（55/60）。14日龄时基因转移率最高，为100%（37/37）。胎龄13、15日龄为97.1%（34/35）。结论：本研究表明基因枪在子宫内基因转移是一种微创技术，是一种有效的将基因转移到小鼠子宫内胎儿的工具

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