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Year : 2020  |  Volume : 3  |  Issue : 1  |  Page : 23-32

CRISPR/Cas9 technology in neurological disorders: An update for clinicians

1 Department of Neurology, All India Institute of Medical Sciences (AIIMS), New Delhi, India
2 Department of Genomics and Molecular Medicine, CSIR-Institute of Genomics and Integrative Biology (CSIR-IGIB), New Delhi, India
3 TAGC-Theories and Approaches of Genomic Complexity, Aix-Marseille University, INSERM, Marseille, France

Correspondence Address:
Prof. Achal K Srivastava
Clinical Neurophysiology Facility, Room No. 60 GF, CN Center, Department of Neurology, All India Institute of Medical Sciences (AIIMS), New Delhi 110029.
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Source of Support: None, Conflict of Interest: None

DOI: 10.4103/AOMD.AOMD_39_19

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Gene therapy has proven its potential in treatment of several human diseases. Most recent method in a long line of genome-editing techniques is Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) system. This CRISPR–Cas9 technology uses a ribonucleic acid (RNA)-guided deoxyribonucleic acid (DNA) endonuclease, Cas9, which induces double-strand breaks (DSBs) in target site. These DSBs are repaired by various cellular DNA repair mechanisms leading to changes in target sites. This revolutionary technique has unraveled several mysteries not only in pathogenesis of several human diseases but also proved its high potential in developing disease models ranging from cell lines to large animals. The number of neurodegenerative disorders linked with mutations has been increasing every day. Several such monogenic disorders provide opportunities for gene therapy using CRISPR–Cas9 method. Translational gap toward developing highly precise and personalized medicine for several neurodegenerative disorders has been reduced by CRISPR–Cas9 technology. Recent advancements in this technique have reduced the adverse effects on targets also. In this review, we have summarized recent achievements of CRISPR–Cas9 technology in common neurological disorders aiming clinicians to understand the technology.

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