Recently, genome editing tools have been extensively used in many biomedical sciences. The gene editing system is applied to modify the dnA sequences in the cellular system to comprehend their physiological response. A developing genome editing technology like clustered regularly short palindromic repeats (CRISPR) is widely used in medical sciences. CRISPR and CRISPR-associated protein 9 (CRISPR/Cas9) system is being exploited to edit any DNA mutations related to inherited ailments to investigate in animals (in vivo) and cell lines (in vitro). Remarkably, CRISPR/Cas9 could be employed to examine treatments of many human genetic diseases such as cystic fibrosis, tyrosinemia, phenylketonuria, muscular dystrophy, Parkinson’s disease, retinoschisis, hemophilia, β-thalassemia and atherosclerosis. Moreover, CRISPR/Cas9 was used for disease resistance such as tuberculosis, Johne’s diseases, chronic enteritis, and brucellosis in animals. Finally, this review discusses existing progress in treating hereditary diseases using CRISPR/Cas9 technology and the high points accompanying obstacles.