Schistosome Transfection Using A Cationic Polymer

描述（由申请人提供）：血吸虫病是一种全球性疾病，感染全球超过2.07亿人。血吸虫病的治疗主要依靠吡喹酮这种药物，这种药物已经使用了30多年。虽然吡喹酮是可用的，但每年有超过25万人死于与疟原虫感染有关的并发症，而且几乎没有正在积极开发的新的抗疟原虫药物。染色体基因组已经被测序，但染色体药物开发的主要障碍是无法有效地操纵染色体基因，导致我们对染色体分子程序的理解有限。我们的长期目标是合理地确定用于治疗血吸虫病的潜在药物靶点。我们计划通过定义可持续发展和可行性所需的监管计划来实现这一目标。这一过程可以通过工具来促进，这些工具可以促进我们在遗传上控制和操纵寄生虫的能力。本研究为通过DNA过表达、蛋白质定位以及RNA干扰策略的应用和发展，将核酸转染到核糖体中以遗传控制核糖体信号系统提供了一种新的方法。它将简化和改进目前的转染方法，并将对寄生虫和寄生虫的分子遗传学产生重大影响。

DESCRIPTION (provided by applicant): Schistosomiasis is a global disease that infects more than 207 million people worldwide. Treatment for schistosomiasis primarily relies on the drug, praziquantel, which has been in use for more than 30 years. Although praziquantel is available, over 250,000 people die each year from complications associated with schistosome infection, and there are few new anti-schistosome drugs undergoing active development. The schistosome genome has been sequenced, but a major hurdle for schistosome drug development has been the inability to efficiently manipulate schistosome genetically resulting in our limited understanding of schistosome molecular programs. Our long-term goals are to rationally identify potential drug targets for use against schistosomiasis. We plan to progress toward this goal by defining the regulatory programs required for schistosome development and viability. This process can be facilitated with tools that promote our ability to genetically modif and manipulate schistosome parasites. This study provides an alternative and new method for the transfection of nucleic acid in schistosomes to genetically control schistosome signaling systems through DNA overexpression, protein localization, and the utilization and the development of RNA interference strategies. It will simplify and improve current methods for transfection and will have a major impact on schistosome, and parasitic worm molecular genetics in general.