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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...
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/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. |
format | Online Article Text |
id | pubmed-9146437 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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