Article

Generation and validation of a human iPSC-derived TDP-43 knockout model for ALS disease modeling

2026-05-02

Abstract excerpt

Nuclear depletion and cytoplasmic aggregation of TDP-43 occur in ∼97% of amyotrophic lateral sclerosis (ALS) cases and disrupt RNA processing through aberrant cryptic exon inclusion. Existing cellular models rely on partial knockdown, TARDBP mutations, or pharmacological stress, each with limitations. Here, we generated homozygous TARDBP -knockout human iPSC lines using CRISPR–Cas9 genome editing and differentia...

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Literature Corpus work: 170efda6-68a3-5831-a8e5-56d4c22dfc77
DOI: 10.64898/2026.04.29.720127

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