Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation Review


Authors: Giacomelli, E.; Vahsen, B. F.; Calder, E. L.; Xu, Y.; Scaber, J.; Gray, E.; Dafinca, R.; Talbot, K.; Studer, L.
Review Title: Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation
Abstract: Neurodegenerative diseases are characterized by progressive cell loss leading to disruption of the structure and function of the central nervous system. Amyotrophic lateral sclerosis (ALS) was among the first of these disorders modeled in patient-specific iPSCs, and recent findings have translated into some of the earliest iPSC-inspired clinical trials. Focusing on ALS as an example, we evaluate the status of modeling neurodegenerative diseases using iPSCs, including methods for deriving and using disease-relevant neuronal and glial lineages. We further highlight the remaining challenges in exploiting the full potential of iPSC technology for understanding and potentially treating neurodegenerative diseases such as ALS. © 2021 Elsevier Inc.
Keywords: pluripotent stem cells; astrocytes; neurodegenerative diseases; microglia; clinical translation; als; disease modeling; ftd; motor neurons: cortical neurons
Journal Title: Cell Stem Cell
Volume: 29
Issue: 1
ISSN: 1934-5909
Publisher: Cell Press  
Date Published: 2022-01-06
Start Page: 11
End Page: 35
Language: English
DOI: 10.1016/j.stem.2021.12.008
PUBMED: 34995492
PROVIDER: scopus
PMCID: PMC8785905
DOI/URL:
Notes: Review -- Export Date: 1 February 2022 -- Source: Scopus
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  1. Lorenz Studer
    223 Studer
  2. Elizabeth L Calder
    10 Calder