An automated platform for the large-scale production of miniaturized neuromuscular organoids

Ziel

Organoids have been developed as advanced 3D cell culture systems that resemble aspects of the in vivo tissues and provide an alternative to study the mechanisms of human disease and identify novel treatments. The last years have witnessed tremendous developments in the field of stem cell and organoid research, but the full potential of these systems remains to be exploited. Two major challenges facing the organoid field are reproducibility and scalability. The manual production of organoids is a labor-intensive and expensive process. The development of cost-effective, fast, and reliable methods is a prerequisite for transferring organoid technologies to the industry for high-throughput approaches. We have recently established a novel complex human neuromuscular organoid (NMO) model from human pluripotent stem cell-derived neuromesodermal progenitors. NMOs self-organize into spinal cord neurons and skeletal muscle compartments that contract by forming functional neuromuscular junctions. The ERC consolidator grant “GPSorganoids” focuses on the generation of position-specific (GPS) NMOs representing distinct spinal cord segments and the use of such NMOs models to study the selective vulnerability of specific spinal cord neurons to neuromuscular diseases like amyotrophic lateral sclerosis and spinal muscular atrophy. The PoC grant goes beyond the scope of our ERC consolidator grant and focuses on the commercialization of the NMO model through the establishment of an automated, reliable, and high throughput production line that could apply to industry settings. Our ultimate goal is to establish NMOs as a leading model in the market for high throughput drug screening approaches and accelerate the development of novel therapies for neuromuscular disorders.

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