Department of Biological and Biomedical Sciences, Glasgow Caledonian University, Glasgow, UK.
Drugs R D. 2011 Sep 1;11(3):227-49. doi: 10.2165/11591280-000000000-00000.
Histone acetyltransferase and histone deacetylase are enzymes responsible for histone acetylation and deacetylation, respectively, in which the histones are acetylated and deacetylated on lysine residues in the N-terminal tail and on the surface of the nucleosome core. These processes are considered the most important epigenetic mechanisms for remodeling the chromatin structure and controlling the gene expression. Histone acetylation is associated with gene activation. Sodium phenylbutyrate is a histone deacetylase inhibitor that has been approved for treatement of urea cycle disorders and is under investigation in cancer, hemoglobinopathies, motor neuron diseases, and cystic fibrosis clinical trials. Due to its characteristics, not only of histone deacetylase inhibitor, but also of ammonia sink and chemical chaperone, the interest towards this molecule is growing worldwide. This review aims to update the current literature, involving the use of sodium phenylbutyrate in experimental studies and clinical trials.
组蛋白乙酰转移酶和组蛋白去乙酰化酶分别负责组蛋白赖氨酸残基 N 端尾部和核小体核心表面的乙酰化和去乙酰化。这些过程被认为是重塑染色质结构和控制基因表达的最重要的表观遗传机制。组蛋白乙酰化与基因激活有关。苯丁酸钠是一种组蛋白去乙酰化酶抑制剂，已被批准用于治疗尿素循环障碍，并正在癌症、血红蛋白病、运动神经元疾病和囊性纤维化临床试验中进行研究。由于其特性，不仅是组蛋白去乙酰化酶抑制剂，还有氨阱和化学伴侣，全世界对这种分子的兴趣正在增长。本综述旨在更新目前的文献，涉及苯丁酸钠在实验研究和临床试验中的应用。
