Modeling ALS with Patient-Derived iPSCs : Recent Advances and Future Potentials

UBC Faculty Research and Publications

Modeling ALS with Patient-Derived iPSCs : Recent Advances and Future Potentials Dawoody Nejad, Ladan; Pioro, Erik P.

Abstract

Amyotrophic lateral sclerosis (ALS) is a terminal complex neurodegenerative disease, with 10–15% of cases being familial and the majority being sporadic with no known cause. There are no animal models for the 85–90% of sporadic ALS cases. More creative, sophisticated models of ALS disease are required to unravel the mysteries of this complicated disease. While ALS patients urgently require new medications and treatments, suitable preclinical in vitro models for drug screening are lacking. Therefore, humanderived induced pluripotent stem cell (hiPSC) technology offers the opportunity to model diverse and unreachable cell types in a culture dish. In this review, we focus on recent hiPSC-derived ALS neuronal and non-neuronal models to examine the research progress of current ALS 2D monocultures, co-cultures, and more complex 3D-model organoids. Despite the challenges inherent to hiPSC-based models, their application to preclinical drug studies is enormous.

Item Metadata

Title	Modeling ALS with Patient-Derived iPSCs : Recent Advances and Future Potentials
Creator	Dawoody Nejad, Ladan; Pioro, Erik P.
Contributor	Djavad Mowafaghian Centre for Brain Health
Publisher	Multidisciplinary Digital Publishing Institute
Date Issued	2025-01-30
Description	Amyotrophic lateral sclerosis (ALS) is a terminal complex neurodegenerative disease, with 10–15% of cases being familial and the majority being sporadic with no known cause. There are no animal models for the 85–90% of sporadic ALS cases. More creative, sophisticated models of ALS disease are required to unravel the mysteries of this complicated disease. While ALS patients urgently require new medications and treatments, suitable preclinical in vitro models for drug screening are lacking. Therefore, humanderived induced pluripotent stem cell (hiPSC) technology offers the opportunity to model diverse and unreachable cell types in a culture dish. In this review, we focus on recent hiPSC-derived ALS neuronal and non-neuronal models to examine the research progress of current ALS 2D monocultures, co-cultures, and more complex 3D-model organoids. Despite the challenges inherent to hiPSC-based models, their application to preclinical drug studies is enormous.
Subject	amyotrophic lateral sclerosis; astrocyte; co-culture; induced pluripotent stem cells; microglia; motor neuron; organoid; preclinical trials
Genre	Article
Type	Text
Language	eng
Date Available	2025-02-27
Provider	Vancouver : University of British Columbia Library
Rights	CC BY 4.0
DOI	10.14288/1.0448141
URI	http://hdl.handle.net/2429/90435
Affiliation	Medicine, Faculty of; Medicine, Department of
Citation	Brain Sciences 15 (2): 134 (2025)
Publisher DOI	10.3390/brainsci15020134
Peer Review Status	Reviewed
Scholarly Level	Faculty; Researcher
Rights URI	https://creativecommons.org/licenses/by/4.0/
Aggregated Source Repository	DSpace

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