Schistosome Transfection Using A Cationic Polymer

描述(申请人提供)：血吸虫病是一种全球性疾病，全世界有超过2.07亿人感染。血吸虫病的治疗主要依靠已经使用了30多年的药物吡喹酮。虽然有吡喹酮可用，但每年有超过25万人死于与血吸虫感染有关的并发症，而且正在积极开发的抗血吸虫新药很少。血吸虫基因组已经测序，但血吸虫药物开发的一个主要障碍是无法有效地从基因上操纵血吸虫，导致我们对血吸虫分子程序的了解有限。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。我们计划通过定义血吸虫发育和生存所需的调控计划来朝着这一目标迈进。这一过程可以通过工具来促进，这些工具可以促进我们从基因上修改和操纵血吸虫寄生虫的能力。本研究通过DNA过表达、蛋白质定位以及RNA干扰策略的利用和发展，为血吸虫核酸转基因调控血吸虫信号系统提供了一种新的途径。它将简化和改进目前的转基因方法，并将对血吸虫和一般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.