Abstract

By introducing recombinant DNA technology, first reported in 1972, biological researchers were able to manipulate DNA molecules to develop new therapeutic strategies. Nowadays, targeted methods for engineering the genome of diverse organisms have provided a powerful tool in the treatment of genetic diseases. Clustered regularly interspaced short palindromic repeats (CRISPR) CRISPR-associated protein 9 (CRISPR/Cas9) technique is one of the newest genome modification tools based on prokaryotic adaptive immune system. The simplicity and flexibility of the CRISPR/Cas9 site-specific nuclease system and its efficiency has led to its widespread use in many biological research areas. In this review, the basis of this technique and its application in the treatment of genetic diseases are explored while highlighting challenges as well as future directions. Derived from a remarkable microbial defense system, CRISPR/Cas9 is used widely as innovative scaffold from basic biology to biotechnology and medicine.

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