CRISPR/ Cas9: A Toolbox with Clinical Potential-Juniper Publishers

The CRISPR/ Cas9 machinery, which is known to provide adaptive immunity to bacteria and archeae, has been proven an efficient tool for genome editing in a wide range of organisms. It is an RNA-guided DNA endonuclease that introduces double-stranded breaks in the target DNA. This compact machinery has been adapted and is being widely used for precise editing and modification of the genome [1]. This review focuses on genome editing using the CRISPR/ Cas9 system for correction of genetic diseases. Here, we review examples from literature where the CRISPR/ Cas9 system has been used for the correction of Duchenne Muscular Dystrophy (DMD), cataract caused due to mutations in the Crygc gene, cystic fibrosis, and sickle cell disease.

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