Department of Physiology and Membrane Biology, University of California School of Medicine, Davis, California, United States.
Biophysics Graduate Group, University of California School of Medicine, Davis, California, United States.
Physiology (Bethesda). 2025 Jan 1;40(1):0. doi: 10.1152/physiol.00029.2024. Epub 2024 Aug 27.
Voltage-gated ion channels (VGICs) are pivotal in regulating electrical activity in excitable cells and are critical pharmaceutical targets for treating many diseases including cardiac arrhythmia and neuropathic pain. Despite their significance, challenges such as achieving target selectivity persist in VGIC drug development. Recent progress in deep learning, particularly diffusion models, has enabled the computational design of protein binders for any clinically relevant protein based solely on its structure. These developments coincide with a surge in experimental structural data for VGICs, providing a rich foundation for computational design efforts. This review explores the recent advancements in computational protein design using deep learning and diffusion methods, focusing on their application in designing protein binders to modulate VGIC activity. We discuss the potential use of these methods to computationally design protein binders targeting different regions of VGICs, including the pore domain, voltage-sensing domains, and interface with auxiliary subunits. We provide a comprehensive overview of the different design scenarios, discuss key structural considerations, and address the practical challenges in developing VGIC-targeting protein binders. By exploring these innovative computational methods, we aim to provide a framework for developing novel strategies that could significantly advance VGIC pharmacology and lead to the discovery of effective and safe therapeutics.
电压门控离子通道(VGICs)在调节可兴奋细胞的电活动中起着至关重要的作用,是治疗许多疾病(包括心律失常和神经性疼痛)的关键药物靶点。尽管它们意义重大,但在 VGIC 药物开发中,仍然存在实现靶标选择性等挑战。最近在深度学习方面的进展,特别是扩散模型,使得仅基于其结构就可以为任何临床相关的蛋白质设计计算蛋白质结合物。这些发展与 VGIC 的实验结构数据的激增相吻合,为计算设计工作提供了丰富的基础。本综述探讨了使用深度学习和扩散方法进行计算蛋白质设计的最新进展,重点介绍了它们在设计调节 VGIC 活性的蛋白质结合物方面的应用。我们讨论了这些方法在计算上设计靶向 VGIC 不同区域的蛋白质结合物的潜在用途,包括孔域、电压感应域和与辅助亚基的接口。我们全面概述了不同的设计场景,讨论了关键的结构考虑因素,并解决了开发靶向 VGIC 的蛋白质结合物的实际挑战。通过探索这些创新的计算方法,我们旨在为开发新的策略提供一个框架,这些策略可以显著推进 VGIC 药理学的发展,并发现有效和安全的治疗方法。