TMPRSS6 作为治疗红细胞生成和铁稳态紊乱的靶点。
TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis.
机构信息
Department of Medicine and Pathology, David Geffen School of Medicine, University of California, Los Angeles, CA, 90095, USA.
Department of Medicine, University of California, Los Angeles, CA, 90095, USA.
出版信息
Adv Ther. 2023 Apr;40(4):1317-1333. doi: 10.1007/s12325-022-02421-w. Epub 2023 Jan 23.
TMPRSS6 is a serine protease highly expressed in the liver. Its role in iron regulation was first reported in 2008 when mutations in TMPRSS6 were shown to be the cause of iron-refractory iron deficiency anemia (IRIDA) in humans and in mouse models. TMPRSS6 functions as a negative regulator of the expression of the systemic iron-regulatory hormone hepcidin. Over the last decade and a half, growing understanding of TMPRSS6 biology and mechanism of action has enabled development of new therapeutic approaches for patients with diseases of erythropoiesis and iron homeostasis.ClinicalTrials.gov identifier NCT03165864.
TMPRSS6 是一种在肝脏中高度表达的丝氨酸蛋白酶。其在铁调节中的作用最早于 2008 年被报道，当时研究表明 TMPRSS6 中的突变是导致人类和小鼠模型中铁难治性缺铁性贫血（IRIDA）的原因。TMPRSS6 作为系统性铁调节激素铁调素表达的负调节剂发挥作用。在过去的十五年中，对 TMPRSS6 生物学和作用机制的深入了解，为治疗红细胞生成和铁稳态疾病的患者提供了新的治疗方法。ClinicalTrials.gov 标识符：NCT03165864。
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