The Switch from Fetal to Adult Hemoglobin
The Switch from Fetal to Adult Hemoglobin
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DOI：
10.1101/cshperspect.a011643
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发表时间：
2013-01-01
影响因子：
5.4
通讯作者：
Orkin, Stuart H.
中科院分区：
文献类型：
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作者：
Sankaran, Vijay G.;Orkin, Stuart H.
胎儿至成人血红蛋白转换和胎儿血红蛋白（HbF）的沉默一直是血液学家长期关注的领域，因为HbF产生的临床诱导具有改善镰状细胞病（SCD）和β-地中海贫血临床症状的巨大希望。在这篇文章中，我们讨论了历史上的尝试，诱导HbF，导致一些治疗方法来管理SCD和β地中海贫血。然后，我们继续讨论最近的分子研究，已经确定了监管机构，包括BCL 11 A，MYB和KLF 1，持有很大的希望，以开发有针对性的和更有效的方法，HbF诱导。我们继续讨论的战略，这种方法可能会发展。这一领域的旧研究可以为未来的研究提供重要的经验教训，旨在制定更有效的HbF诱导策略，因此，我们按时间顺序涵盖了这一领域在过去四十年中发展所完成的工作。
The fetal-to-adult hemoglobin switch and silencing of fetal hemoglobin (HbF) have been areas of long-standing interest among hematologists, given the fact that clinical induction of HbF production holds tremendous promise to ameliorate the clinical symptoms of sickle cell disease (SCD) and beta-thalassemia. In this article, we discuss historic attempts to induce HbF that have resulted in some therapeutic approaches to manage SCD and beta-thalassemia. We then go on to discuss how more recent molecular studies that have identified regulators, including BCL11A, MYB, and KLF1, hold great promise to develop targeted and more effective approaches for HbF induction. We go on to discuss strategies by which such approaches may be developed. Older studies in this field can provide important lessons for future studies aimed at developing more effective strategies for HbF induction, and we therefore chronologically cover the work accomplished as this field has evolved over the course of the past four decades.