The Application of Human iPSCs in Neurological Diseases: From Bench to Bedside

Figure 1

Applications of human iPSCs and iPSC derived neural cells in neurological diseases. In principle, induced pluripotent stem cells (iPSCs) are generated from somatic cells by reprogramming and gaining the capacity to self-renew indefinitely as well as the ability to differentiate into cells of different lineages. Up to date, the main applications of iPSC products in neurological diseases have included disease modeling, drug screening, and cell replacement therapy, for which the successful reprogramming of somatic cells to iPSCs is the most fundamental step. By generating human-animal chimeras, 3D culture systems, and cerebral organoids with disease specific iPSCs and iPSC derived neural cells, researchers can have various systems for disease modeling and drug screening. By inducing disease relevant cell types into wild type (WT) iPSCs, researchers and clinicians can have normal cells for cell replacement therapy. Recently, the rapid development of genome editing technology, including TALEN, ZFN, and CRISPR, makes it possible for diseases with defined genetic disorders to switch phenotype between WT and mutant at the single cell level, providing a new strategy to create syngeneic WT cells for cell replacement therapy [103]. Overall, human iPSCs have a great potential for clinical applications in neurological diseases.