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Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective

A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has previously been established that the induction of fetal hemoglobin (HbF) production in significant amounts can reduce the severity of the clinical course in diseases such as...

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Autores principales: Bou-Fakhredin, Rayan, De Franceschi, Lucia, Motta, Irene, Cappellini, Maria Domenica, Taher, Ali T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9227505/
https://www.ncbi.nlm.nih.gov/pubmed/35745672
http://dx.doi.org/10.3390/ph15060753
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author Bou-Fakhredin, Rayan
De Franceschi, Lucia
Motta, Irene
Cappellini, Maria Domenica
Taher, Ali T.
author_facet Bou-Fakhredin, Rayan
De Franceschi, Lucia
Motta, Irene
Cappellini, Maria Domenica
Taher, Ali T.
author_sort Bou-Fakhredin, Rayan
collection PubMed
description A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has previously been established that the induction of fetal hemoglobin (HbF) production in significant amounts can reduce the severity of the clinical course in diseases such as β-thalassemia and sickle cell disease (SCD). While the induction of HbF using lentiviral and genome-editing strategies has been made possible, they present limitations. Meanwhile, progress in the use of pharmacologic agents for HbF induction and the identification of novel HbF-inducing strategies has been made possible as a result of a better understanding of γ-globin regulation. In this review, we will provide an update on all current pharmacological inducer agents of HbF in β-thalassemia and SCD in addition to the ongoing research into other novel, and potentially therapeutic, HbF-inducing agents.
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spelling pubmed-92275052022-06-25 Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective Bou-Fakhredin, Rayan De Franceschi, Lucia Motta, Irene Cappellini, Maria Domenica Taher, Ali T. Pharmaceuticals (Basel) Review A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has previously been established that the induction of fetal hemoglobin (HbF) production in significant amounts can reduce the severity of the clinical course in diseases such as β-thalassemia and sickle cell disease (SCD). While the induction of HbF using lentiviral and genome-editing strategies has been made possible, they present limitations. Meanwhile, progress in the use of pharmacologic agents for HbF induction and the identification of novel HbF-inducing strategies has been made possible as a result of a better understanding of γ-globin regulation. In this review, we will provide an update on all current pharmacological inducer agents of HbF in β-thalassemia and SCD in addition to the ongoing research into other novel, and potentially therapeutic, HbF-inducing agents. MDPI 2022-06-16 /pmc/articles/PMC9227505/ /pubmed/35745672 http://dx.doi.org/10.3390/ph15060753 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Bou-Fakhredin, Rayan
De Franceschi, Lucia
Motta, Irene
Cappellini, Maria Domenica
Taher, Ali T.
Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
title Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
title_full Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
title_fullStr Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
title_full_unstemmed Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
title_short Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
title_sort pharmacological induction of fetal hemoglobin in β-thalassemia and sickle cell disease: an updated perspective
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9227505/
https://www.ncbi.nlm.nih.gov/pubmed/35745672
http://dx.doi.org/10.3390/ph15060753
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