Mirza Ahmed, Menhart Nick
Department of Biological, Chemical and Physical Sciences, Illinois Institute of Technology, 3101 South Dearborn, Chicago, IL 60616, USA.
Biochim Biophys Acta. 2008 Sep;1784(9):1301-9. doi: 10.1016/j.bbapap.2008.05.010. Epub 2008 Jun 6.
Dystrophin is a rod shaped protein consisting of amino- and carboxy-terminal binding domains linked by a large central rod composed of 24 homologous copies of the STR motif and 4 non-homologous regions termed hinges. These hinges are proposed to confer local flexibility; conversely, the tacit implication is that the STR regions away from the hinges are comparatively rigid. This, and the repeating nature of this rod, has contributed to the view that the STR region of the rod is uniform and monolithic. However, we have produced various 2 STR fragments, chosen to have high and low alpha-helix content at their junctions with each other, and show that they exhibit markedly different stabilities. In contrast to a related protein, spectrin, these differences are not correlated with the calculated helicity, but appear to be an intrinsic property of the motifs themselves. A full understanding of how these properties vary along the length of the rod has implications for the engineering of these rods regions in exon skipping and minidystrophin therapies.
肌营养不良蛋白是一种杆状蛋白,由氨基末端和羧基末端结合结构域组成,两者通过一个大的中央杆相连,该中央杆由STR基序的24个同源拷贝和4个称为铰链的非同源区域组成。这些铰链被认为赋予局部柔韧性;相反,不言而喻的含义是远离铰链的STR区域相对刚性。这一点以及该杆的重复性质,导致了这样一种观点,即杆的STR区域是均匀且整体的。然而,我们制备了各种2个STR片段,选择它们在彼此连接处具有高和低α-螺旋含量,并表明它们表现出明显不同的稳定性。与相关蛋白血影蛋白不同,这些差异与计算出的螺旋度无关,而似乎是这些基序本身的固有特性。全面了解这些特性如何沿杆的长度变化,对于在外显子跳跃和微型肌营养不良蛋白疗法中对这些杆区域进行工程设计具有重要意义。