Regulation of Fetal Hemoglobin Production in Humans.

总结：胎儿血红蛋白在出生时表达减少是镰状细胞和β-地中海贫血综合征临床表现的定义机制。因此，预防或逆转该过程代表了治疗这些疾病的主要临床目标。该实验室的一个持续的兴趣涉及研究胎儿血红蛋白的表达和调节，造血干细胞经历分化成为红系祖细胞和前体细胞。有几种方法用于操纵胎儿血红蛋白的表达，包括信号转导、化学诱导和胎儿珠蛋白基因的遗传调节。目前的焦点来自临床转录组研究，该研究确定了let-7 microRNA在调节胎儿血红蛋白中的潜在参与。目前正在进行研究，以探索let-7 microRNA相关的生物学，目标是开发血红蛋白疾病的新疗法。

Summary: Decreased expression of fetal hemoglobin around the time of birth represents the defining mechanism in the clinical manifestation of sickle-cell and beta-thalassemia syndromes. Therefore, prevention or reversal of that process represents a major clinical goal for the treatment of those diseases. An ongoing interest of the laboratory involves the study of fetal hemoglobin expression and regulation as hematopoietic stem cells undergo differentiation to become erythroid progenitor and precursor cells. Several approaches are used to manipulate the expression of fetal hemoglobin including signal transduction, chemical induction, and genetic modulation of the fetal globin gene. A current focus arose from clinical transcriptome studies that identified the potential involvement of let-7 microRNA in the regulation of fetal hemoglobin. Studies are now underway to explore let-7 microRNA related biology with a goal of developing novel therapies for hemoglobin disorders.