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Development of plasmid DNA-based gene transfer

机译：基于质粒DNA的基因转移的发展

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Gene therapy based on ultrasound with microbubbles offers a novel approach for the prevention and treatment of variety of diseases. The major development of gene transfer has importantly contributed to intense investigation of the potential of gene therapy in cancer or cardiovascular medicine. The amazing advances in molecular biology have provided a dramatic improvement of the technology that is necessary to transfer target genes into somatic cells. Gene transfer methods have been surprisingly improved. In fact, some of them (retroviral vectors, adenoviral vectors or liposome based vectors, etc.) have been used in the clinical trials already. But some severe side effects were reported in clinical gene therapy using such viral, so people desire safe and efficient clinical gene therapy. Recently, ultrasound-mediated gene transfer has been reported to augment the transfection efficiency and facilitate local gene expression. Interestingly, gene transfer into the fetal central nervous system was successfully achieved by intrauterine injection with microbubble-enhanced ultrasound. Compared to other viral vectors, there are some theoretical advantages including safety, simplicity of preparation, and local gene transfer. Thus, we focused on the development of gene transfer using naked plasmid DNA with an ultrasound or microbubble-enhanced ultrasound method.
机译：基于超声与微泡的基因治疗为预防和治疗多种疾病提供了一种新颖的方法。基因转移的重大发展为深入研究癌症或心血管医学中基因治疗的潜力做出了重要贡献。分子生物学的惊人进步为将靶基因转移到体细胞中所必需的技术提供了巨大的进步。基因转移方法已经令人惊讶地得到改善。实际上，其中一些（逆转录病毒载体，腺病毒载体或基于脂质体的载体等）已用于临床试验。但是据报道，在使用这种病毒的临床基因治疗中有一些严重的副作用，因此人们希望获得安全有效的临床基因治疗。近来，已经报道了超声介导的基因转移以提高转染效率并促进局部基因表达。有趣的是，通过宫腔内注射微泡增强超声成功地将基因转移至胎儿中枢神经系统。与其他病毒载体相比，在理论上有一些优势，包括安全性，制备简便性和局部基因转移。因此，我们专注于使用裸质粒DNA结合超声或微泡增强超声方法进行基因转移的开发。

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