Stem Cell-Based Disease Models for Inborn Errors of Immunity.
hematopoietic stem cells
immune deficiency
inborn errors of immunity
induced pluripotent stem cells
primary immune deficiency
Journal
Cells
ISSN: 2073-4409
Titre abrégé: Cells
Pays: Switzerland
ID NLM: 101600052
Informations de publication
Date de publication:
30 12 2021
30 12 2021
Historique:
received:
01
12
2021
revised:
23
12
2021
accepted:
29
12
2021
entrez:
11
1
2022
pubmed:
12
1
2022
medline:
31
3
2022
Statut:
epublish
Résumé
The intrinsic capacity of human hematopoietic stem cells (hHSCs) to reconstitute myeloid and lymphoid lineages combined with their self-renewal capacity hold enormous promises for gene therapy as a viable treatment option for a number of immune-mediated diseases, most prominently for inborn errors of immunity (IEI). The current development of such therapies relies on disease models, both in vitro and in vivo, which allow the study of human pathophysiology in great detail. Here, we discuss the current challenges with regards to developmental origin, heterogeneity and the subsequent implications for disease modeling. We review models based on induced pluripotent stem cell technology and those relaying on use of adult hHSCs. We critically review the advantages and limitations of current models for IEI both in vitro and in vivo. We conclude that existing and future stem cell-based models are necessary tools for developing next generation therapies for IEI.
Identifiants
pubmed: 35011669
pii: cells11010108
doi: 10.3390/cells11010108
pmc: PMC8750661
pii:
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Swiss National Science Foundation
ID : P500PB_203146 / 1
Pays : Switzerland
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