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首页> 外文期刊>AIMS Cell and Tissue Engineering >Modeling rare pediatric neurogenetic disorders with IPSCs
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Modeling rare pediatric neurogenetic disorders with IPSCs

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Intensive research has been performed to identify the pathological mechanisms of manypediatric neurogenetic disorders and to identify potential therapeutic targets. Although research intomany pediatric neurological disorders has provided tremendous insight into the mechanisms ofdisease, effective treatments remain elusive. A significant impediment to progress has been a lack ofthorough disease models. Transgenic/knockout animal models have been very valuable indetermining the mechanisms of many neurogenetic disorders; however, these models cannot alwaysmimic human-specific pathology and can be inadequate in representing human pathogenesis. Thiscan be especially true for diseases of the nervous system. Alternatively, human patient-derivednervous tissue can be dangerous to acquire and difficult to propagate. The development ofpatient-derived induced pluripotent stem cells (IPSCs) has given researchers a fresh means ofmodeling these disorders with renewable human cells that can be used to generate neurons and glia.IPSCs are somatic cells that are reprogrammed back to a pluripotent stage, which can provide anunlimited source of human cells possessing patient-specific genetic mutations. Their potential to bedifferentiated into any cell type enables them to be a flexible platform to investigate neurogeneticdisease. Of course, efficient methods for differentiating IPSCs into homogeneous populations ofsomatic cells must be established to provide the “disease-in-a-dish” systems. We will discuss thecurrent methods for generating IPSC-derived neural cells to model pediatric neurogenetic disorders,as well as provide examples of the disorders that have been studied that include severalneurodevelopmental and neurodegenerative disorders (Rett syndrome, spinal muscular atrophy,hereditary spastic paraplegias, and leukodystrophies). In addition, we provide examples on howpatient-specific neural cells can be used in therapeutic development with high-throughput drugscreening platforms or with correction via genome editing.

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