Background: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) associated Cas9 (CRISPR/Cas9) system has been using from last few years in the field of biomedical research. Here, applications of CRISPR–Cas9 technology in congenital disorders, current and future directions are reviewed and discussed.

Methods: This non–systematic review was prepared using search engine: PubMed, Google Scholar and Medline for articles published from 2005 through 2017, using the following keywords: genome editing, CRISPR/Cas9, genetic disorders, DMD, Cystic fibrosa, hemophilia, Thalassemia etc.

Results: The defect in gene sequences can be corrected by deletion or insertion using CRISPR/Cas9 and this property has made this gene editing tool for wider applications.

Conclusion: CRISPR/Cas9 genome editing could be used in the future to correct inherited mutations and it is expected that in near future, genome editing technologies would help to accelerate the therapeutic directions towards understanding of molecular mechanism of disease at gene level.