ALIU, Hesat and ADEMI, Elmedina (2022) CRISPR/CAS GENE THERAPY. Journal of Natural Sciences and Mathematics of UT, 7 (13-14). pp. 209-213. ISSN 2671-3039

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Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system. Recently, Cas9/CRISPR has been reported to successfully induce targeted gene disruption and homologous recombination in both prokaryotic and eukaryotic cells with higher efficiency compared with ZFN and TALEN. Additionally, it is easier to design guide sequence and easy to use for Cas9/CRISPR system. This novel technology will be of great potential for application in both research field and clinical trials. This Review describes the development of technologies based on nuclease-deactivated Cas9, termed dCas9, for RNA-guided genomic transcription regulation, both by repression through CRISPR interference (CRISPRi) and by activation through CRISPR activation (CRISPRa).

Item Type: Article
Uncontrolled Keywords: Gene therapy, CRISPR/Cas9, ZNF, Talen
Subjects: Q Science > QH Natural history > QH301 Biology
Divisions: Faculty of Medicine, Health and Life Sciences > School of Biological Sciences
Depositing User: Unnamed user with email
Date Deposited: 31 Oct 2022 14:28
Last Modified: 31 Oct 2022 14:29

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