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Poster

Modeling ALS Using Patient-Derived iPSCs: A Human-Relevant Platform for Disease Research and Therapeutic Discovery

A 10-day iPSC differentiation protocol generating spinal motor neurons that replicate TDP-43 mislocalization, validated across sporadic and familial ALS patient lines with compound rescue data.

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Inside This Poster

Examine a patient-derived iPSC platform that reproduces ALS pathology in human spinal motor neurons and demonstrates compound-driven correction of TDP-43 mislocalization within 10 days of differentiation.

  • Motor neurons differentiated from healthy and ALS donor iPSCs expressed pan-neuronal marker TUBB3 (95%+), cholinergic marker ChAT (70%+), and motor neuron marker HB9 (70%+) by Day 10.
  • Sporadic ALS patient-derived motor neurons showed significantly elevated cytoplasmic TDP-43 localization compared to healthy controls, confirmed by One-way ANOVA (p<0.0001) across two sporadic and one familial ALS line.
  • Treatment with Tubastatin A, CK1 inhibitor, Sephin1, and Edaravone each significantly reduced cytoplasmic TDP-43 accumulation in ALS motor neurons and restored nuclear localization.