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Article

Rapid and Robust Multi-Phenotypic Assay System for ALS Using Human iPS Cells with Mutations in Causative Genes.

International journal of molecular sciences - 10 Apr 2023

Kondo Tosho, Ebinuma Ihori, Tanaka Hirotaka, Nishikawa Yukitoshi, Komiya Takaki, Ishikawa Mitsuru, Okano Hideyuki

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a major life-threatening disease caused by motor neuron degeneration. More effective treatments through drug discovery are urgently needed. Here, we established an effective high-throughput screening system using induced pluripotent stem cells (iPSCs). Using a Tet-On-dependent transcription factor expression system carried on the PiggyBac vector, motor neurons were...

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PMID: 37108151
DOI: 10.3390/ijms24086987
PMCID: PMC10138792

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