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A novel formulation for controlled release of plasmid DNA: minipellet

机译：一种新型的质粒DNA控制释放的制剂：微米池

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Gene delivery via plasmid DNA(pDNA) is superior to that via viral vectors in terms of safety and ease feasibility of mass production. Despite these advantages, however, the use of pDNA has been precluded by its low-level and short-term expression. Although liposome vectors have been shown to be effective to enhance the translation efficiency and increase the geen expresison, the duration of gene expression could not be extended since these vectors translate the pDNA immediately after administration. On the other hand, the stability of the pDNA contained in the vectors and the storage conditions of the vectors have been investigated scarcely. Therefore, a practical pDNA delivery method applicable for clinical use is desired. We have developed a collagen-based injectable sustained release formualtion, the Minipellet (MP~1), and MP containing interferon has been filed to MHW. In this study, we adapted the MP as a novel formulation for controlled release of pDNA, that allows prolonged release and expression of pDNA~2). In this report, we report on the development of pDNA-MP from the following viewpoints:1) stability and stabilization of the pDNA, 2) controlled release of pDNA, 3) sustained release of pDNA in vivo, 4) extension of period of protein expression.
机译：通过质粒DNA（PDNA）的基因递送优于通过病毒载体在安全性和容易的批量生产的可行性方面优异。然而，尽管这些优点，但是通过其低水平和短期表达缺乏PDNA的使用。虽然已经显示脂质体载体可以有效地增强翻译效率并增加Geen Expresison，但是由于这些载体在给药后立即转化PDNA，因此不能延长基因表达的持续时间。另一方面，已经研究了载体中包含的PDNA的稳定性和载体的储存条件。因此，期望适用于临床使用的实际PDNA递送方法。我们开发了一种基于胶原蛋白的可注射持续释放Formualtion，Minipellet（MP〜1）和含有温干扰素的MP已提交给MHW。在该研究中，我们将MP调整为用于控制释放的PDNA的新型制剂，其允许延长释放和表达PDNA〜2）。在本报告中，我们从以下观点报告了PDNA-MP的发展：1）PDNA的稳定性和稳定性，2）PDNA的受控释放，3）体内PDNA的持续释放，4）蛋白质期间的延长表达。

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