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成人红细胞中胎儿血红蛋白生成的异质性。

Heterogeneity of fetal hemoglobin production in adult red blood cells.

机构信息

Division of Hematology, The Children's Hospital of Philadelphia; Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.

出版信息

Curr Opin Hematol. 2021 May 1;28(3):164-170. doi: 10.1097/MOH.0000000000000640.

Abstract

PURPOSE OF REVIEW

Small amounts of fetal hemoglobin can be expressed in a subset of adult red blood cells called F-cells. This review examines the potential mechanisms and clinical implications of the heterogeneity of fetal hemoglobin expression.

RECENT FINDINGS

Although the heterocellular nature of fetal hemoglobin expression in adult red blood cells has been noted for over 70 years, the molecular basis of this phenomenon has been unclear. Recent discoveries of novel regulators of fetal hemoglobin as well as technological advances have shed new light on these cells.

SUMMARY

Fetal hemoglobin reactivation in adult red blood cells through genetic or pharmacological approaches can involve both increasing the number of F-cells and cellular fetal hemoglobin content. New technologies enable the study and eventually the improvement of these parameters in patients with sickle cell disease and β-thalassemia.

摘要

目的综述

少量胎儿血红蛋白可在称为 F 细胞的一部分成人红细胞中表达。本综述探讨了胎儿血红蛋白表达异质性的潜在机制和临床意义。

最近的发现

尽管胎儿血红蛋白在成人红细胞中的异质性已被注意到超过 70 年,但这种现象的分子基础尚不清楚。最近发现了胎儿血红蛋白的新型调节因子以及技术进步,为这些细胞提供了新的认识。

总结

通过遗传或药物方法在成人红细胞中重新激活胎儿血红蛋白可能涉及增加 F 细胞的数量和细胞内胎儿血红蛋白含量。新技术使我们能够研究并最终改善镰状细胞病和β-地中海贫血患者的这些参数。

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本文引用的文献

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Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease.用 治疗镰状细胞病的转录后基因沉默。
N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5.
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Fetal hemoglobin in sickle cell anemia.镰状细胞贫血中的胎儿血红蛋白。
Blood. 2020 Nov 19;136(21):2392-2400. doi: 10.1182/blood.2020007645.
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Recent Advances in the Treatment of Sickle Cell Disease.镰状细胞病治疗的最新进展
Front Physiol. 2020 May 20;11:435. doi: 10.3389/fphys.2020.00435. eCollection 2020.
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Current and future gene therapies for hemoglobinopathies.用于血红蛋白病的当前和未来基因疗法。
Curr Opin Hematol. 2020 May;27(3):149-154. doi: 10.1097/MOH.0000000000000581.

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