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Recent Advances in Genome-Engineering Strategies

期刊

GENES
卷 14, 期 1, 页码 -

出版社

MDPI
DOI: 10.3390/genes14010129

关键词

CRISPR-Cas systems; dCas9; nucleases; ZFNs; TALENs; CAR-T cells; gene therapy; gene knockout; genome editing

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In October 2020, Emmanuelle Charpentier and Jennifer A. Doudna were awarded the chemistry Nobel Prize for their discovery of CRISPR-Cas9, a promising genome-editing tool. The identification of CRISPR arrays and cas genes has led to the development of strategies for precise DNA editing, which have been widely used in biological and medical research.
In October 2020, the chemistry Nobel Prize was awarded to Emmanuelle Charpentier and Jennifer A. Doudna for the discovery of a new promising genome-editing tool: the genetic scissors of CRISPR-Cas9. The identification of CRISPR arrays and the subsequent identification of cas genes, which together represent an adaptive immunological system that exists not only in bacteria but also in archaea, led to the development of diverse strategies used for precise DNA editing, providing new insights in basic research and in clinical practice. Due to their advantageous features, the CRISPR-Cas systems are already employed in several biological and medical research fields as the most suitable technique for genome engineering. In this review, we aim to describe the CRISPR-Cas systems that have been identified among prokaryotic organisms and engineered for genome manipulation studies. Furthermore, a comprehensive comparison between the innovative CRISPR-Cas methodology and the previously utilized ZFN and TALEN editing nucleases is also discussed. Ultimately, we highlight the contribution of CRISPR-Cas methodology in modern biomedicine and the current plethora of available applications for gene KO, repression and/or overexpression, as well as their potential implementation in therapeutical strategies that aim to improve patients' quality of life.

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