Departments of †Chemical Engineering and ‡Molecular Biosciences, University of Texas at Austin , Austin, Texas 78712, United States.
Mol Pharm. 2017 Oct 2;14(10):3269-3280. doi: 10.1021/acs.molpharmaceut.7b00322. Epub 2017 Sep 18.
Understanding protein stability is central to combatting protein aggregation diseases and developing new protein therapeutics. At the high concentrations often present in biological systems, purified proteins can exhibit undesirable high solution viscosities and poor solubilities mediated by short-range electrostatic and hydrophobic protein-protein interactions. The interplay between protein amino acid sequence, protein structure, and solvent conditions to minimize protein-protein interactions is key to designing well-behaved pharmaceutical proteins. However, theoretical approaches have yet to yield a general framework to address these problems. Here, we analyzed the high concentration behavior of superfolder GFP (sfGFP) and two supercharged sfGFP variants engineered to have formal charges of -18 or +15. Under low cosolute conditions, sfGFP and the -18 variant formed a gel or phase separated at ∼10 mg/mL. Under conditions that screen surface charges, including formulations with high histidine or high NaCl concentrations, all three variants attained concentrations up to 250 mg/mL with moderate viscosities. Moreover, all three variants exhibited very similar viscosity-concentration profiles over this range. This effect was not mimicked by high sugar concentrations that exert excluded-volume effects without shielding charge. Collectively, these data demonstrate that charge shielding neutralizes not only long-range electrostatic interactions but also, surprisingly, short-range electrostatic effects due to surface charge anisotropy. This work shows that supercharged sfGFP behavior under high ionic strength is largely determined by particle geometry, a conclusion that is supported by colloid models and may be applicable to pharmaceutically relevant proteins.
理解蛋白质稳定性对于对抗蛋白质聚集疾病和开发新的蛋白质治疗方法至关重要。在生物系统中经常存在的高浓度下,纯化的蛋白质可能表现出不理想的高溶液粘度和差的溶解度,这是由短程静电和疏水蛋白质-蛋白质相互作用介导的。蛋白质氨基酸序列、蛋白质结构和溶剂条件之间的相互作用,以最小化蛋白质-蛋白质相互作用,是设计行为良好的药物蛋白质的关键。然而,理论方法尚未产生一个通用的框架来解决这些问题。在这里,我们分析了超折叠 GFP(sfGFP)和两种带超高电荷的 sfGFP 变体在高浓度下的行为,这两种变体的形式电荷分别为-18 或+15。在低共溶剂条件下,sfGFP 和-18 变体在约 10mg/mL 时形成凝胶或相分离。在屏蔽表面电荷的条件下,包括含有高组氨酸或高 NaCl 浓度的配方,所有三种变体在 250mg/mL 的浓度下都具有适中的粘度。此外,所有三种变体在这个范围内都表现出非常相似的粘度-浓度曲线。高糖浓度不能模拟这种效果,高糖浓度虽然具有排除体积效应,但不能屏蔽电荷。总的来说,这些数据表明,电荷屏蔽不仅中和了长程静电相互作用,而且令人惊讶的是,还中和了由于表面电荷各向异性引起的短程静电效应。这项工作表明,在高离子强度下带超高电荷的 sfGFP 行为主要取决于颗粒几何形状,胶体模型支持这一结论,并且可能适用于药物相关的蛋白质。