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  • Current Issues in Molecular Biology is published by MDPI from Volume 43 Issue 1 (2021). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Caister Press.
  • Review
  • Open Access

7 September 2017

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

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1
State Key Laboratory of Agriculture Microbiology, Huazhong Agricultural University, Wuhan, China
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Institute of Biochemistry and Biotechnology, University of Veterinary and Animal Sciences, Lahore, Pakistan
3
Key Laboratory of Agricultural Animal Genetics, Breeding and Reproduction of the Ministry of Education, College of Animal Science and Technology, Huazhong Agricultural University, Wuhan, China
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Author to whom correspondence should be addressed.

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.

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