Sickle cell disease: challenges and advances in drug discovery

Quimica nova

Chin, Chung Man¹  Santos, Jean Leandro dos¹ 

[1] Universidade Estadual Paulista Júlio de Mesquita Filho, Araraquara, Brasil

关键词: Sickle Cell Disease;    drug discovery;    molecular modification.;   

DOI  :  10.1590/S0100-40422012000400025

学科分类：化学（综合）

来源: Sociedade Brasileira de Quimica

【 摘 要 】

Sickle Cell Disease (SCD) is a disease characterized by a punctual mutation (GTG - GAG) in the sixth codon of the gamma globin gene leading to a substitution of glutamic acid by a valine in the β chain of hemoglobin. Despite the huge progress on the molecular knowledge of the disease in recent years, few therapeutic resources were developed. Currently, the treatment is mainly done with the anticancer agent hydroxyurea. This review summarizes current knowledge about possible targets and new approaches to the discovery new compounds to treat the symptoms of SCD.

【 授权许可】

CC BY