Article

CRISPR/Cas9-mediated generation of a <i>de novo C9ORF72</i> knock-in isogenic iPSC cell bank to model ALS and FTD

2025-11-13

Abstract excerpt

<h4>Background</h4> The pathogenic G 4 C 2 repeat expansion in the C9ORF72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Studies focused on delineating the underlying perturbed mechanisms resulting from this genetic mutation are often confounded by the heterogeneity present in current disease models, such as patient-derived iPSC lines, with esti...

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Literature Corpus work: 52b4b313-d863-5305-9e15-db89f6247901
DOI: 10.1101/2025.11.12.687564

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