Organ-on-a-chip technologies to study neuromuscular disorders: possibilities, limitations, and future hopes

MEDIZINISCHE GENETIK(2021)

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摘要
Neuromuscular disorders are a heterogeneous group of diseases ranging from mild to devastating phenotypes depending on the disorder's origin. Pathophysiologies for many of these disorders are not fully understood and efficient therapies are urgently needed. Recent advances in the field of induced pluripotent stem cells and organ-on-a-chip technologies have brought enormous improvement in modeling neuromuscular diseases. Even complex units, like the neuromuscular junction, can now be built, enabling researchers to study each component of the motor unit by itself or interacting with others, allowing the identification of disease mechanisms. This article aims to introduce these new modeling systems to study neuromuscular disorders and the possibilities of organ on-a-chip platforms to shed light on disease pathologies and their use for therapy development.
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关键词
disease modeling, induced pluripotent stem cells, neuromuscular disorders, neuromuscular junction, organ-on-a-chip
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