Journal
CURRENT PHARMACEUTICAL BIOTECHNOLOGY
Volume 18, Issue 13, Pages 1038-1054Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1389201019666180209120533
Keywords
CRISPR-Cas systems; guide RNA (gRNA); gene editing; mutation; off-target effect; single-strand specific DNA and RNA endonucleases
Funding
- Biotechnology Development Council of the Islamic Republic of Iran [950609]
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Background: RNA-guided endonuclease as a versatile genome editing technology opened new windows in various fields of biology. The simplicity of this revolutionary technique provides a promising future for its application in a broad range of approaches from functional annotation of genes to diseases, to genetic manipulation and gene therapy. Besides the site-specific activity of Cas9 endonuclease, the unintended cleavage known as off-target effect is still a major challenge for this genome editing technique. Methods: Various strategies have been developed to resolve this bottleneck including development of new softwares for designing optimized guide RNA (gRNA), engineering Cas9 enzyme, improvement in off-target detection assays, etc. Results: This review dedicated to discuss on methods that have been used for optimizing Cas9, specificity with the aim of improving this technology for therapeutic applications. Conclusion: In addition, the applications and novel breakthroughs in the field of CRISPR technology will be described.
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