iPSC-based research in ALS precision medicine.

Mazzini, Letizia; De Marchi, Fabiola · Cell Stem Cell · 2023

basic_science · Level V

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Abstract

Clinical trials in amyotrophic lateral sclerosis (ALS) are challenged by the lack of pre-clinical models and biomarkers of disease onset and progression. In this issue, Morimoto et al. use induced pluripotent stem cell (iPSC)-derived motor neurons from patients with ALS to study therapeutic mechanisms of ropinirole in a clinical trial and identify treatment responders.

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