LeMaster David M, Hernandez Griselda
Wadsworth Center, New York State Department of Health, Empire State Plaza, Albany, New York, 12201, USA; Department of Biomedical Sciences, School of Public Health, University at Albany - SUNY, Empire State Plaza, Albany, New York, 12201, USA.
Curr Mol Pharmacol. 2015;9(1):5-26. doi: 10.2174/1874467208666150519113146.
Among the 22 FKBP domains in the human genome, FKBP12.6 and the first FKBP domains (FK1) of FKBP51 and FKBP52 are evolutionarily and structurally most similar to the archetypical FKBP12. As such, the development of inhibitors with selectivity among these four FKBP domains poses a significant challenge for structure-based design. The pleiotropic effects of these FKBP domains in a range of signaling processes such as the regulation of ryanodine receptor calcium channels by FKBP12 and FKBP12.6 and steroid receptor regulation by the FK1 domains of FKBP51 and FKBP52 amply justify the efforts to develop selective therapies. In contrast to their close structural similarities, these four FKBP domains exhibit a substantial diversity in their conformational flexibility. A number of distinct conformational transitions have been characterized for FKBP12 spanning timeframes from 20 s to 10 ns and in each case these dynamics have been shown to markedly differ from the conformational behavior for one or more of the other three FKBP domains. Protein flexibilitybased inhibitor design could draw upon the transitions that are significantly populated in only one of the targeted proteins. Both the similarities and differences among these four proteins valuably inform the understanding of how dynamical effects propagate across the FKBP domains as well as potentially how such intramolecular transitions might couple to the larger scale transitions that are central to the signaling complexes in which these FKBP domains function.
在人类基因组的22个FKBP结构域中,FKBP12.6以及FKBP51和FKBP52的首个FKBP结构域(FK1)在进化和结构上与典型的FKBP12最为相似。因此,开发在这四个FKBP结构域之间具有选择性的抑制剂对基于结构的设计构成了重大挑战。这些FKBP结构域在一系列信号传导过程中具有多效性,例如FKBP12和FKBP12.6对兰尼碱受体钙通道的调节以及FKBP51和FKBP52的FK1结构域对类固醇受体的调节,充分证明了开发选择性疗法的努力是合理的。与它们紧密的结构相似性形成对比的是,这四个FKBP结构域在构象灵活性方面表现出很大的差异。已经对FKBP12在从20秒到10纳秒的时间范围内的许多不同构象转变进行了表征,并且在每种情况下,这些动力学都已显示出与其他三个FKBP结构域中的一个或多个的构象行为明显不同。基于蛋白质灵活性的抑制剂设计可以利用仅在一种目标蛋白中大量存在的转变。这四种蛋白质之间的异同对于理解动力学效应如何在FKBP结构域中传播以及这种分子内转变可能如何与这些FKBP结构域发挥作用的信号复合物的核心大规模转变耦合非常有价值。