Genome editing involves the insertion, deletion, modification or replacement of DNA in the genome with the help of synthetic nucleases, the “molecular scissors”. Targeting genome alteration of stem cells in disease models is a prerequisite for utilizing the full potential of stem cells. Genome edited human iPSCs can be employed to study gene function, drug and chemical screens. Many genome-editing techniques have been launched to improve the efficiency and speed of the development of stem cells for human disease models.