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Implications of hematopoietic stem cells heterogeneity for gene therapies
Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allogeneic HSCT bear considerable risks for the patient s...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8455331/ https://www.ncbi.nlm.nih.gov/pubmed/33589780 http://dx.doi.org/10.1038/s41434-021-00229-x |
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author | Epah, Jeremy Schäfer, Richard |
author_facet | Epah, Jeremy Schäfer, Richard |
author_sort | Epah, Jeremy |
collection | PubMed |
description | Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allogeneic HSCT bear considerable risks for the patient such as non-engraftment, or graft-versus host disease. Transplanting gene modified autologous HSCs is a promising approach not only for inherited blood/immune cell diseases, but also for the acquired immunodeficiency syndrome. However, there is emerging evidence for substantial heterogeneity of HSCs in situ as well as ex vivo that is also observed after HSCT. Thus, HSC gene modification concepts are suggested to consider that different blood disorders affect specific hematopoietic cell types. We will discuss the relevance of HSC heterogeneity for the development and manufacture of gene therapies and in exemplary diseases with a specific emphasis on the key target HSC types myeloid-biased, lymphoid-biased, and balanced HSCs. |
format | Online Article Text |
id | pubmed-8455331 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Nature Publishing Group UK |
record_format | MEDLINE/PubMed |
spelling | pubmed-84553312021-10-07 Implications of hematopoietic stem cells heterogeneity for gene therapies Epah, Jeremy Schäfer, Richard Gene Ther Review Article Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allogeneic HSCT bear considerable risks for the patient such as non-engraftment, or graft-versus host disease. Transplanting gene modified autologous HSCs is a promising approach not only for inherited blood/immune cell diseases, but also for the acquired immunodeficiency syndrome. However, there is emerging evidence for substantial heterogeneity of HSCs in situ as well as ex vivo that is also observed after HSCT. Thus, HSC gene modification concepts are suggested to consider that different blood disorders affect specific hematopoietic cell types. We will discuss the relevance of HSC heterogeneity for the development and manufacture of gene therapies and in exemplary diseases with a specific emphasis on the key target HSC types myeloid-biased, lymphoid-biased, and balanced HSCs. Nature Publishing Group UK 2021-02-15 2021 /pmc/articles/PMC8455331/ /pubmed/33589780 http://dx.doi.org/10.1038/s41434-021-00229-x Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . |
spellingShingle | Review Article Epah, Jeremy Schäfer, Richard Implications of hematopoietic stem cells heterogeneity for gene therapies |
title | Implications of hematopoietic stem cells heterogeneity for gene therapies |
title_full | Implications of hematopoietic stem cells heterogeneity for gene therapies |
title_fullStr | Implications of hematopoietic stem cells heterogeneity for gene therapies |
title_full_unstemmed | Implications of hematopoietic stem cells heterogeneity for gene therapies |
title_short | Implications of hematopoietic stem cells heterogeneity for gene therapies |
title_sort | implications of hematopoietic stem cells heterogeneity for gene therapies |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8455331/ https://www.ncbi.nlm.nih.gov/pubmed/33589780 http://dx.doi.org/10.1038/s41434-021-00229-x |
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