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Treating Genetic Disorders Using State-Of-The-Art Technology.

Authors :
Jamal M
Ullah A
Ahsan M
Tyagi R
Habib Z
Khan FA
Rehman K
Source :
Current issues in molecular biology [Curr Issues Mol Biol] 2018; Vol. 26, pp. 33-46. Date of Electronic Publication: 2017 Sep 07.
Publication Year :
2018

Abstract

CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR associated Protein 9), basically a bacterial immune system is now widely applicable to engineer genomes of a number of cells and organisms because of its simplicity and robustness. In research avenue the system has been optimized to regulate gene expression, modify epigenome and edit target locus. These applications make CRISPR/Cas9, a technology of choice to edit disease causing mutations as well as the epigenome more efficiently than ever before. Meanwhile its application in in vivo and ex vivo cells is encouraging the scientific community for more vigorous gene therapy and in clinical setups for therapeutic genome editing. Here we review the recent advances that CRISPR-Cas9 mediated genome editing has achieved and is reported in previous studies and address the challenges associated with it.

Details

Language :
English
ISSN :
1467-3045
Volume :
26
Database :
MEDLINE
Journal :
Current issues in molecular biology
Publication Type :
Academic Journal
Accession number :
28879854
Full Text :
https://doi.org/10.21775/cimb.026.033