Caister Academic Press

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

Muhammad Jamal, Arif Ullah, Muhammad Ahsan, Rohit Tyagi, Zeshan Habib, Faheem Ahmad Khan and Khaista Rehman
from: The CRISPR/Cas System: Emerging Technology and Application (Edited by: Muhammad Jamal). Caister Academic Press, U.K. (2017) Pages: 33-46.


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 read more ...
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