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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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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. |
format | Online Article Text |
id | pubmed-9227505 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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