CRISPR genome editing and its medical applications

Mohammad-Reza Mahmoudian-Sani,Gholamreza Farnoosh,Ali Mahdavinezhad,Massoud Saidijam

doi:10.1080/13102818.2017.1406823

Abstract

ABSTRACTGenome editing has always been a challenging area as a means to provide more efficient ways to create a meaningful change in the genome. Today, the CRISPR (clustered regularly interspaced short palindromic repeat) restoration system is considered as one of the suitable and promising options for genome editing. This system has many advantages compared to the previous gene-editing methods developed in this area. Compared to the previous systems, CRISPR can deactivate or eliminate a gene without interfering with intracellular mechanisms. The system can be used in the treatment of diseases and in related research with identifying the performance of defective genes in these diseases. CRISPR has more potentials and applications compared to previous systems. Among these applications, we can note the use of CRISPR in understanding genetic and epigenetic diseases such as cancer. Study of cancer by the CRISPR system is done by two approaches: turning off the oncogenes and turning on the tumour suppressor genes. According to the exact capability of CRISPR, this system can also be used to create exact mutations in different cell lines to model the cancers. This type of modeling can lead to a better understanding of cancer and the ability to develop effective drugs.

Highlights

The clustered regularly interspaced short palindromic repeat (CRISPR) system was first discovered as a defence system in Escherichia coli against viruses
In international meetings and conferences, numerous promises are made regarding the possibilities for treatment and fixing of genetic defects in human embryos using the CRISPR technique
Genetic manipulation in humans may have been merely a theoretical concept; but the CRISPR technique has made a large step forward to achieving this. This technique has triggered ethical disputes, it is a fact that we already live in a CRISPR world

Summary

Introduction

The clustered regularly interspaced short palindromic repeat (CRISPR) system was first discovered as a defence system in Escherichia coli against viruses. This advanced technology has the potential to reform and change the genetic pool in a society, and to make basic changes in the healthcare system, the food, drug, agriculture and all industries related to biological sciences. Two methods of zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) were used for this purpose [1]. These methods had limitations due to high costs, the difficulty of endonucleases system design, and the low performance of precise cutting [2–5]

Background

Findings

Conclusions