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Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease

In the last decade, research on pathophysiology and therapeutic solutions for β-thalassemia (BThal) and sickle cell disease (SCD) has been mostly focused on the primary erythroid defect, thus neglecting the study of hematopoietic stem cells (HSCs) and bone marrow (BM) microenvironment. The quality a...

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Autores principales: Aprile, Annamaria, Sighinolfi, Silvia, Raggi, Laura, Ferrari, Giuliana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9146437/
https://www.ncbi.nlm.nih.gov/pubmed/35631417
http://dx.doi.org/10.3390/ph15050592
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author Aprile, Annamaria
Sighinolfi, Silvia
Raggi, Laura
Ferrari, Giuliana
author_facet Aprile, Annamaria
Sighinolfi, Silvia
Raggi, Laura
Ferrari, Giuliana
author_sort Aprile, Annamaria
collection PubMed
description In the last decade, research on pathophysiology and therapeutic solutions for β-thalassemia (BThal) and sickle cell disease (SCD) has been mostly focused on the primary erythroid defect, thus neglecting the study of hematopoietic stem cells (HSCs) and bone marrow (BM) microenvironment. The quality and engraftment of HSCs depend on the BM microenvironment, influencing the outcome of HSC transplantation (HSCT) both in allogeneic and in autologous gene therapy settings. In BThal and SCD, the consequences of severe anemia alter erythropoiesis and cause chronic stress in different organs, including the BM. Here, we discuss the recent findings that highlighted multiple alterations of the BM niche in BThal and SCD. We point out the importance of improving our understanding of HSC biology, the status of the BM niche, and their functional crosstalk in these disorders towards the novel concept of combined therapies by not only targeting the genetic defect, but also key players of the HSC–niche interaction in order to improve the clinical outcomes of transplantation.
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spelling pubmed-91464372022-05-29 Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease Aprile, Annamaria Sighinolfi, Silvia Raggi, Laura Ferrari, Giuliana Pharmaceuticals (Basel) Review In the last decade, research on pathophysiology and therapeutic solutions for β-thalassemia (BThal) and sickle cell disease (SCD) has been mostly focused on the primary erythroid defect, thus neglecting the study of hematopoietic stem cells (HSCs) and bone marrow (BM) microenvironment. The quality and engraftment of HSCs depend on the BM microenvironment, influencing the outcome of HSC transplantation (HSCT) both in allogeneic and in autologous gene therapy settings. In BThal and SCD, the consequences of severe anemia alter erythropoiesis and cause chronic stress in different organs, including the BM. Here, we discuss the recent findings that highlighted multiple alterations of the BM niche in BThal and SCD. We point out the importance of improving our understanding of HSC biology, the status of the BM niche, and their functional crosstalk in these disorders towards the novel concept of combined therapies by not only targeting the genetic defect, but also key players of the HSC–niche interaction in order to improve the clinical outcomes of transplantation. MDPI 2022-05-11 /pmc/articles/PMC9146437/ /pubmed/35631417 http://dx.doi.org/10.3390/ph15050592 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
Aprile, Annamaria
Sighinolfi, Silvia
Raggi, Laura
Ferrari, Giuliana
Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease
title Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease
title_full Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease
title_fullStr Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease
title_full_unstemmed Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease
title_short Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease
title_sort targeting the hematopoietic stem cell niche in β-thalassemia and sickle cell disease
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9146437/
https://www.ncbi.nlm.nih.gov/pubmed/35631417
http://dx.doi.org/10.3390/ph15050592
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