Pluripotent Stem Cells for Modeling Motor Neuron Diseases
作者： Delphine Bohl
DOI: 10.1007/978-3-319-33270-3_5
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摘要: Motoneuron disorders (MNDs) are a group of degenerative diseases characterized by muscular weakness and muscle atrophy that result most often in the death patients. Today, there is no treatment despite decades clinical trials. One reason failures these trials may be anatomic physiologic differences exist between humans rodent models MNDs. With recent discovery human induced pluripotent stem cell (iPSC) technology, new hopes arise to generate cellular for In this chapter, I will focus on two common infantile adult MNDs, respectively: spinal (SMA) amyotrophic lateral sclerosis (ALS). describe iPSC generated from patients with various forms each disorder specific defects observed iPS-derived motoneurons order validate identify phenotypes could targets future therapies SMA ALS
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