Inborn errors of metabolism: Lessons from iPSC models.
CRISPR/Cas9
Disease modeling
Human induced pluripotent stem cells
Lysosomal storage disorders
Reprogramming
Targeted genome edition
Journal
Reviews in endocrine & metabolic disorders
ISSN: 1573-2606
Titre abrégé: Rev Endocr Metab Disord
Pays: Germany
ID NLM: 100940588
Informations de publication
Date de publication:
12 2021
12 2021
Historique:
accepted:
30
06
2021
pubmed:
10
7
2021
medline:
15
3
2022
entrez:
9
7
2021
Statut:
ppublish
Résumé
The possibility of reprogramming human somatic cells to pluripotency has opened unprecedented opportunities for creating genuinely human experimental models of disease. Inborn errors of metabolism (IEMs) constitute a greatly heterogeneous class of diseases that appear, in principle, especially suited to be modeled by iPSC-based technology. Indeed, dozens of IEMs have already been modeled to some extent using patient-specific iPSCs. Here, we review the advantages and disadvantages of iPSC-based disease modeling in the context of IEMs, as well as particular challenges associated to this approach, together with solutions researchers have proposed to tackle them. We have structured this review around six lessons that we have learnt from those previous modeling efforts, and that we believe should be carefully considered by researchers wishing to embark in future iPSC-based models of IEMs.
Identifiants
pubmed: 34241766
doi: 10.1007/s11154-021-09671-z
pii: 10.1007/s11154-021-09671-z
pmc: PMC8724155
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1189-1200Informations de copyright
© 2021. The Author(s).
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