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Annals of Hematology

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04.01.2021 | Review Article

Erythrocyte microRNAs: a tiny magic bullet with great potential for sickle cell disease therapy

verfasst von: Henu Kumar Verma, Yashwant Kumar Ratre, L. V. K. S. Bhaskar, Raffaella Colombatti

Erschienen in: Annals of Hematology | Ausgabe 3/2021

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Abstract

Sickle cell disease (SCD) is a severe hereditary blood disorder caused by a mutation of the beta-globin gene, which results in a substantial reduction in life expectancy. Many studies are focused on various novel therapeutic strategies that include re-activation of the γ-globin gene. Among them, expression therapy caused by the fetal hemoglobin (HbF) at a later age is highly successful. The induction of HbF is one of the dominant genetic modulators of the hematological and clinical characteristics of SCD. In fact, HbF compensates for the abnormal beta chain and has an ameliorant effect on clinical complications. Erythropoiesis is a multi-step process that involves the proliferation and differentiation of a small population of hematopoietic stem cells and is affected by several factors, including signaling pathways, transcription factors, and small non-coding RNAs (miRNAs). miRNAs play a regulatory role through complex networks that control several epigenetic mechanisms as well as the post-transcriptional regulation of multiple genes. In this review, we briefly describe the current understanding of interactions between miRNAs, their molecular targets, and their regulatory effects in HbF induction in SCD.

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Titel: Erythrocyte microRNAs: a tiny magic bullet with great potential for sickle cell disease therapy
verfasst von: Henu Kumar Verma
Yashwant Kumar Ratre
L. V. K. S. Bhaskar
Raffaella Colombatti
Publikationsdatum: 04.01.2021
Verlag: Springer Berlin Heidelberg
Erschienen in: Annals of Hematology / Ausgabe 3/2021
Print ISSN: 0939-5555
Elektronische ISSN: 1432-0584
DOI: https://doi.org/10.1007/s00277-020-04390-y

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