Figure 1.

Applications of iPSCs for disease modeling and autologous cell-based therapy. Disease-specific iPSCs can be generated from patients with inherited blood diseases. A panel of disease-specific iPSCs and their derivatives enable high-throughput screening assay against the library of hundreds of thousand compounds. This approach represents a powerful tool for elucidating disease mechanisms and developing new drugs. Alternatively, the genome editing technology can be employed to correct genetic mutations followed by directed differentiation; the gene-corrected iPSC-derived hematopoietic stem cells (HSCs) or other mature blood cells can be transplanted or transfused to the same patient.