Advances and challenges in modeling inherited peripheral neuropathies using iPSCs

Jonas Van Lent, Robert Prior, Gonzalo Pérez Siles, Anthony N. Cutrupi, Marina L. Kennerson, Tim Vangansewinkel, Esther Wolfs, Bipasha Mukherjee-Clavin, Zachary Nevin, Luke Judge, Bruce Conklin, Henna Tyynismaa, Alex J. Clark, David L. Bennett, Ludo Van Den Bosch, Mario Saporta, Vincent Timmerman

Forskningsoutput: TidskriftsbidragÖversiktsartikelPeer review

Sammanfattning

Inherited peripheral neuropathies (IPNs) are a group of diseases associated with mutations in various genes with fundamental roles in the development and function of peripheral nerves. Over the past 10 years, significant advances in identifying molecular disease mechanisms underlying axonal and myelin degeneration, acquired from cellular biology studies and transgenic fly and rodent models, have facilitated the development of promising treatment strategies. However, no clinical treatment has emerged to date. This lack of treatment highlights the urgent need for more biologically and clinically relevant models recapitulating IPNs. For both neurodevelopmental and neurodegenerative diseases, patient-specific induced pluripotent stem cells (iPSCs) are a particularly powerful platform for disease modeling and preclinical studies. In this review, we provide an update on different in vitro human cellular IPN models, including traditional two-dimensional monoculture iPSC derivatives, and recent advances in more complex human iPSC-based systems using microfluidic chips, organoids, and assembloids.

Originalspråkengelska
TidskriftExperimental and Molecular Medicine
Volym56
Sidor (från-till)1348-1364
Antal sidor17
ISSN1226-3613
DOI
StatusPublicerad - 2024
MoE-publikationstypA2 Granska artikel i en vetenskaplig tidskrift

Bibliografisk information

Publisher Copyright:
© The Author(s) 2024.

Vetenskapsgrenar

  • 1182 Biokemi, cell- och molekylärbiologi

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