FIGURE 1.

The route to apply iPSCs and iN technology for neurodegenerative disease modeling and drug discovery. To establish an in vitro disease model, the first step is to obtain fibroblasts from a diseased person. Upon overexpression of certain transcription factors, fibroblasts can be directly or indirectly reprogrammed to neurons. Depending on research purposes, the fibroblasts from original iPSCs can be modified by gene editing (CRISPR CAS9). The neurons generated would be applied for drug screening according to the disease phenotype. Subsequently, the best candidates could then be used for pre-clinical studies on drug toxicity, bio-availability, pharmacology, and metabolism in animals. Finally, the potential drug would be used for clinical studies and therapy. DP: disease person.