Fig. 1

Application of patient derived iPSCs for disease modeling and cell therapy (A) Establishment of patient derived iPSCs (or disease iPSCs) allows the production of somatic cells with pathogenic phenotypes (i.e., “Disease-in-a-dish”), which would be ultimate cell source to study the molecular mechanism to underlying disease and to screen small molecules to revert the phenotypes. Gene correction is critical to produce the isogenic control iPSCs to enable the precise comparison to avoid the variation from the different genetic background. (B) Autologous cell therapy from the patient with a genetic disease is achieved by gene correction of pathogenic mutations from disease iPSCs. The functionally intact somatic cells from the edited iPSCs serve as ideal cell source for reconstitution of specific organ with disease phenotype. Created with BioRender.com