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 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.

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Author:Jeremy EpahGND, Richard SchäferORCiDGND
Parent Title (English):Gene therapy
Publisher:Nature Publ. Group
Place of publication:London
Document Type:Article
Date of Publication (online):2021/02/15
Date of first Publication:2021/02/15
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2022/10/24
Tag:Cell biology; Diseases; Gene therapy; Hematopoietic stem cells; Heterogeneity; Lineage; Subpopulation
Page Number:14
First Page:528
Last Page:541
Open Access funding enabled and organized by Projekt DEAL.
Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Licence (German):License LogoCreative Commons - Namensnennung 4.0