Lapdesf - Laboratório de Pesquisa e Desenvolvimento de Fármacos, Departamento de Fármacos e Medicamentos, Universidade Estadual Paulista-UNESP, Rodovia Araraquara Jaú Km., Araraquara, SP, Brazil.
Curr Med Chem. 2011;18(15):2339-58. doi: 10.2174/092986711795656117.
Sickle Cell Disease (SCD) is one of the most prevalent hematological diseases in the world. SCD is a genetic disease characterized by punctual mutation that basis on the exchange of glutamic acid to valine in a beta chain of hemoglobin. In deoxygenated state, the interaction among the beta chains leads to hemoglobin polymerization carrying out to deformation of cytoskeleton structure of red blood cells to a sickle shape. Currently, the treatment is performed with the antineoplasic drug hydroxyurea. This review summarizes current knowledge about possible targets and the approaches to discover new compounds to treat the SCD symptoms. Drug design based on therapeutical application and molecular modifications strategies will be discussed.
镰状细胞病(SCD)是世界上最常见的血液系统疾病之一。SCD 是一种遗传性疾病,其特征在于血红蛋白β链上谷氨酸被替换为缬氨酸的点突变。在脱氧状态下,β链之间的相互作用导致血红蛋白聚合,从而使红细胞的细胞骨架结构变形为镰状。目前,治疗方法是使用抗肿瘤药物羟基脲。本综述总结了关于可能的靶点和发现新化合物治疗 SCD 症状的方法的最新知识。将讨论基于治疗应用和分子修饰策略的药物设计。