Journal of Genetic Syndromes & Gene Therapy
Open Access

Abstract

Application of CRISPR/Cas9 Genome Editing in Genetic Disorders: A Systematic Review Up to Date

Vivek K Pandey, Anima Tripathi, Ravi Bhushan, Akhtar Ali and Pawan K Dubey

Genetic diseases in human are associated with congenital disorders and phenotypic traits. A single mutation in a gene can cause physical or mental problems, and sometimes both. Some diseases can be lethal, and there are still no cures for many of them. Socioeconomic burden of rare genetic diseases are increasing worldwide that have been tried to cure using various methods. However, they were not very successful till now. Genome editing technologies over the past few years is providing fast and effective tool to precisely manipulate the genome at specific locations. 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. CRISPR/Cas9 has advantages in terms of clinical applicability to treat genetic diseases like DMD, Hemophilia, β-Thalassemia and cystic fibrosis etc. and even in some cases this tool has already been successfully applied. Nevertheless, developed technologies for addition or deletion of genes have made notable progress in last few years and demonstrate some promising clinical results. However, several challenges still remain. Here, the latest applications of CRISPR-Cas9 technology in genetic disorders, current challenges and future directions are reviewed and discussed.