iPSC-Based Disease Modeling of Childhood Neurogenetic Disorders

Description

This template was adapted from the original submission. Edits were made to enhance scientific accuracy, optimal usability and/or to meet industry-leading design standards for science communication. Human induced pluripotent stem cells (iPSC) are an unprecedented tool for in vitro disease modeling. First, somatic cells from patients and healthy controls are reprogrammed to iPSC and differentiated into the relevant neuronal cell type. Then, these neurons can be used for in vitro assays to elucidate the molecular mechanism causing the disease. Finally, different drugs can be pre-clinically tested on patient-derived cells with the ultimate goal of clinical translation.

Acknowledgements

References

Barral et al., . (2016) Utility of Induced Pluripotent Stem Cells for the Study and Treatment of Genetic Diseases: Focus on Childhood Neurological Disorders. Front Mol Neurosci. https://doi.org/10.3389/fnmol.2016.00078

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