Santini V, Gozzini A, Scappini B, Grossi A, Rossi Ferrini P
Department of Hematology, University of Florence, Firenze, Italy.
Leuk Lymphoma. 2001 Jul;42(3):275-89. doi: 10.3109/10428190109064584.
n-Butyric acid and its "polymorphic" derivatives have been largely but somehow "blindly" studied in oncology and in red cell diseases with consistent results through decades indicating a strong maturative effect determined by enhancement of gene transcription. Although these effects have been observed mainly in vitro, the relative absence of systemic toxicity of butyrates render these compounds appealing as specific therapeutic agents. More interestingly, their specific mechanism of action, i.e. inhibition of histone deacetylase and de-repression of transcription represents at present an unique tool for diseases such as acute leukemias which are characterised by a disregulation of co-repressors and co-activators of gene transcription. More insight into specificity and modalities of action of different butyrate derivatives may be a guarantee for excellent tailored antileukemic therapy in the future.
正丁酸及其“多晶型”衍生物在肿瘤学和红细胞疾病领域已得到广泛研究，但在一定程度上是“盲目”进行的。几十年来，研究结果一致表明，它们通过增强基因转录产生强大的成熟效应。尽管这些效应主要在体外观察到，但丁酸盐相对缺乏全身毒性，这使得这些化合物作为特定治疗剂具有吸引力。更有趣的是，它们的具体作用机制，即抑制组蛋白脱乙酰酶和解除转录抑制，目前是治疗急性白血病等疾病的独特工具，这些疾病的特征是基因转录的共抑制因子和共激活因子失调。更深入了解不同丁酸盐衍生物的特异性和作用方式，可能为未来出色的个性化抗白血病治疗提供保障。
