Clinical and experimental applications of sodium phenylbutyrate.
Clinical and experimental applications of sodium phenylbutyrate.
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DOI：
10.2165/11591280-000000000-00000
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发表时间：
2011-09-01
期刊：
影响因子：
3
通讯作者：
Palmieri B
中科院分区：
文献类型：
--
作者：
Iannitti T;Palmieri B
组蛋白乙酰化转移酶和组蛋白去乙酰化酶分别负责组蛋白乙酰化和去乙酰化，组蛋白在N末端尾部的赖氨酸残基和核小体核心表面的赖氨酸残基上乙酰化和去乙酰化。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酶抑制剂，已被批准用于治疗尿素循环障碍，目前正在进行癌症、血红蛋白疾病、运动神经元病和囊性纤维化的临床试验。由于其不仅具有组蛋白脱乙酰酶抑制剂的特性，而且还具有氨沉和化学伴侣的特性，因此在世界范围内对该分子的研究越来越受到重视。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使用。
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.