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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 NMPs to neurons (Tuj1) and muscles (Desmin) 

in 2D cell culture conditions


In vitro differentiation of NMPs to spinal cord neurons (Tuj1) and muscles

(Myosin Skeletal Fast) in 3D cell culture conditions.

Neuromuscular Organoid day 100

The Gouti Lab is actively involved in iMed, which includes five other Helmholtz centers in the Research Field Health besides the MDC. The program aims to interlink and coordinate the centers’ research activities in the field of personalized medicine and, through collaboration with local clinical partners, to promote the rapid transfer of research results into clinical practice.

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