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Modeling human neuromuscular diseases using patient induced pluripotent stem cells


The last years, remarkable progress has been made in the generation of spinal cord motor neurons (MNs) from human PSCs and induced PSCs. However, the ability of these MNs to connect and control human skeletal muscle remains uncertain. We use human PSC derived NMP cells to generate in parallel spinal cord neurons and muscle cells in 2D and 3D cell culture systems. This allows us to study the origin of human neuromuscular diseases and the contribution of either lineage (neural – muscle) to its pathology. We focus on Spinal Muscular Atrophy (SMA), Amyotrophic Lateral Sclerosis (ALS), muscular dystrophies (MD) and Myasthenia gravis (MG). 

In vitro differentiation of hPSCs to neurons (Tuj1) and skeletal muscles (MyoSF)  that form functional NMJs in adherent culture conditions (Urzi et al, Nature Communications, 2023)


In vitro differentiation of NMPs to spinal cord neurons (Tuj1) and muscles
(Myosin Skeletal Fast) in 3D cell culture conditions.
Neuromuscular Organoid day 100

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