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A human iPSC-based platform to screen therapeutics for ALS using specific and robust phenotypic assays

Human iPSC-derived motor neuron assays for ALS drug discovery and therapeutic screening
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ALS poster

This poster presents Ncardia’s human iPSC-derived motor neuron platform for amyotrophic lateral sclerosis (ALS) research, using a CRISPR-engineered TDP-43 mutation to model key disease-associated phenotypes. The platform combines high-content imaging, molecular analysis and electrophysiological measurements to support the evaluation of potential ALS therapeutics.

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  • How TDP-43 mislocalisation and pathological aggregation are quantified in human iPSC-derived motor neurons using high-content imaging

  • How STMN2 protein reduction and abnormal STMN2 splicing provide measurable readouts of ALS-related molecular pathology

  • How multi-electrode array (MEA) recordings and neurofilament-L measurements capture changes in neuronal activity and disease-associated phenotypes

  • How 384-well miniaturised assays support compound screening, alongside proof-of-concept gene therapy experiments demonstrating a reduction in TDP-43 aggregation

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Whether you are investigating ALS disease mechanisms, evaluating therapeutic candidates or developing phenotypic screening assays, Ncardia’s human iPSC-based ALS platform provides multiple complementary readouts for studying disease-associated changes and assessing potential treatments.

 

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