Figure 1.

(A) iPSC-based cell therapy for inheritable RBC disorders. Patient fibroblasts can be obtained from skin biopsy or other tissues, followed by reprogramming with distinct set of factors to establish patient-specific iPSCs. These iPSCs bearing genetic mutations will be corrected with gene editing approaches to produce disease-free iPSCs. The corrected iPSCs are autologous and able to be differentiated into hematopoietic stem cells in vitro for therapeutic purposes. (B) Summary of iPSC from anemic patients. K, KLF4; O, OCT4; S, SOX2; L, L-MYC; M, c-MYC.