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- Title
CRISPR/CAS GENE THERAPY.
- Authors
ALIU, Hesat; ADEMI, Elmedina
- Abstract
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).
- Subjects
GENE therapy; CRISPRS; EUKARYOTIC cells; IMMUNE system; CLINICAL trials
- Publication
Journal of Natural Sciences & Mathematics (JNSM), 2022, Vol 7, Issue 13/14, p214
- ISSN
2545-4072
- Publication type
Article