The application of induced pluripotent stem cell technology for therapeutic purposes. Patient-derived somatic cells can be isolated through tissue biopsies and converted into induced pluripotent stem cells (iPSCs) through reprogramming. From there, iPSCs can be expanded into suitable quantities before differentiation into desired tissue types for transplantation purposes. Gene targeting of patient-derived iPSCs can also be done through homologous recombination or via gene-editing nucleases to correct genetic mutations. Upon successful modification, the genetically corrected iPSCs can then be expanded, differentiated and transplanted back into the patient for cell therapy. iPSCs from patients harboring genetic diseases can similarly be used as an in vitro disease model to study disease pathogenesis, or for drug development and screening. Data gained through the study of disease-specific cell culture models will enable the identification of critical molecular and cellular pathways in disease development, and allow for the formulation of effective treatment strategies.