NCYTE Cell 2 Motorneurons DAPI PAX7 MAP2 Nestin.tif RGB green

Motor Neuron diseases

We offer a diverse porfolio of motor neuron disease models designed to accelerate preclinical discovery. This includes patient‑derived iPSC lines, gene‑edited models carrying disease-associated mutations, and matched isogenic controls.

Using small-molecule patterning combined with NGN2-driven differentiation, we reliably generate Hb9/MNX1-positive lower motor neurons. These neurons are extensively characterized and can be cultured as monolayers or in co-culture with human iPSC-derived muscle cells to study neuromuscular junction formation, stability, and degeneration (Guerra San Juan et al., Neurobiology of Disease, 2025).

Through systematic functional profiling, we have esthablished a high‑quality reference dataset of motor neuron phenotypes, enabling rapid, reproducible, and quantitative evaluation of therapeutic candidates.

ALS-Relevant Phenotypic Analyses

Our platform enables multiparametric characterization of ALS phenotypes, including:

Motor neuron survival and degeneration kinetics
Neuronal network activity and excitability
Axonal outgrowth, degeneration, and regeneration
Axonal transport of mitochondria, RNA granules and other organelles
Neuromuscular junction formation and maintenance
Synaptic connectivity and vesicle release
TDP-43 mislocalization, aggregation phenotypes and TDP-43 driven splicing defects
Cellular stress responses and stress granule dynamics

These readouts allow quantitative assessment of disease mechanisms, target engagement, and functional rescue by therapeutic compounds.

Available IPSC disease models

Extensively validated control iPSC lines
IPSC lines containing UNC13A risk SNP variants that enhance cryptic exon inclusion upon TDP-43 loss
Patient-derived iPSC lines from familial or sporadic ALS cases
Gene-edited lines with patient mutations and matched isogenic controls (TARDBP, FUS, GRN, KIF5A, and MAPT).