Department of Medicinal Chemistry, College of Pharmacy, University of Florida, Gainesville, FL 32610, USA.
Department of Anatomy and Cell Biology, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
Cell Chem Biol. 2023 Nov 16;30(11):1421-1435.e12. doi: 10.1016/j.chembiol.2023.07.010. Epub 2023 Aug 11.
HDAC3 and HDAC8 have critical biological functions and represent highly sought-after therapeutic targets. Because histone deacetylases (HDACs) have a very conserved catalytic domain, developing isozyme-selective inhibitors remains challenging. HDAC3/8 also have deacetylase-independent activity, which cannot be blocked by conventional enzymatic inhibitors. Proteolysis-targeting chimeras (PROTACs) can selectively degrade a target enzyme, abolishing both enzymatic and scaffolding function. Here, we report a novel HDAC3/8 dual degrader YX968 that induces highly potent, rapid, and selective degradation of both HDAC3/8 without triggering pan-HDAC inhibitory effects. Unbiased quantitative proteomic experiments confirmed its high selectivity. HDAC3/8 degradation by YX968 does not induce histone hyperacetylation and broad transcriptomic perturbation. Thus, histone hyperacetylation may be a major factor for altering transcription. YX968 promotes apoptosis and kills cancer cells with a high potency in vitro. YX968 thus represents a new probe for dissecting the complex biological functions of HDAC3/8.
HDAC3 和 HDAC8 具有重要的生物学功能,是备受关注的治疗靶点。由于组蛋白去乙酰化酶(HDACs)具有非常保守的催化结构域,因此开发同工酶选择性抑制剂仍然具有挑战性。HDAC3/8 还具有去乙酰化酶非依赖性活性,无法被传统的酶抑制剂阻断。蛋白水解靶向嵌合体(PROTACs)可以选择性地降解靶酶,从而消除酶和支架功能。在这里,我们报告了一种新型的 HDAC3/8 双降解剂 YX968,它可以诱导 HDAC3/8 的高效、快速和选择性降解,而不会引发全 HDAC 抑制作用。无偏定量蛋白质组学实验证实了其高选择性。YX968 对 HDAC3/8 的降解不会诱导组蛋白过度乙酰化和广泛的转录组扰动。因此,组蛋白过度乙酰化可能是改变转录的一个主要因素。YX968 在体外具有很高的效价,能促进细胞凋亡并杀死癌细胞。因此,YX968 代表了一种用于剖析 HDAC3/8 复杂生物学功能的新探针。