Deu Edgar
Chemical Biology Approaches to Malaria Laboratory, The Francis Crick Institute, London, UK.
FEBS J. 2017 Aug;284(16):2604-2628. doi: 10.1111/febs.14130. Epub 2017 Jul 3.
Malaria is a devastating parasitic disease affecting half of the world's population. The rapid emergence of resistance against new antimalarial drugs, including artemisinin-based therapies, has made the development of drugs with novel mechanisms of action extremely urgent. Proteases are enzymes proven to be well suited for target-based drug development due to our knowledge of their enzymatic mechanisms and active site structures. More importantly, Plasmodium proteases have been shown to be involved in a variety of pathways that are essential for parasite survival. However, pharmacological rather than target-based approaches have dominated the field of antimalarial drug development, in part due to the challenge of robustly validating Plasmodium targets at the genetic level. Fortunately, over the last few years there has been significant progress in the development of efficient genetic methods to modify the parasite, including several conditional approaches. This progress is finally allowing us not only to validate essential genes genetically, but also to study their molecular functions. In this review, I present our current understanding of the biological role proteases play in the malaria parasite life cycle. I also discuss how the recent advances in Plasmodium genetics, the improvement of protease-oriented chemical biology approaches, and the development of malaria-focused pharmacological assays, can be combined to achieve a robust biological, chemical and therapeutic validation of Plasmodium proteases as viable drug targets.
疟疾是一种极具破坏力的寄生虫病,影响着全球一半的人口。包括青蒿素类疗法在内的新型抗疟药物耐药性迅速出现,使得开发具有新作用机制的药物变得极为紧迫。蛋白酶是一类酶,鉴于我们对其酶促机制和活性位点结构的了解,已证明它们非常适合基于靶点的药物开发。更重要的是,疟原虫蛋白酶已被证明参与了多种对寄生虫生存至关重要的途径。然而,在抗疟药物开发领域,药理学方法而非基于靶点的方法一直占据主导地位,部分原因是在基因水平上有力地验证疟原虫靶点存在挑战。幸运的是,在过去几年中,在开发用于改造寄生虫的高效遗传方法方面取得了重大进展,包括几种条件性方法。这一进展最终不仅使我们能够在基因层面验证必需基因,还能研究它们的分子功能。在这篇综述中,我阐述了我们目前对蛋白酶在疟原虫生命周期中所起生物学作用的理解。我还讨论了如何将疟原虫遗传学的最新进展、面向蛋白酶的化学生物学方法的改进以及针对疟疾的药理学检测方法的开发结合起来,以实现对疟原虫蛋白酶作为可行药物靶点进行有力的生物学、化学和治疗学验证。