| Literature DB >> 29714581 |
Jorge E Simón1, Ángel S Rodríguez1, Nelson Santiago Vispo1.
Abstract
In the last few decades, genomic manipulation has made significant progress as a result of the development of recombinant DNA technologies; however, more often than not, these techniques have been costly and labor intensive. In contrast, recently developed next-generation sequencing (NGS) technologies have provided a cheaper, faster, and easier process to study genomics. In particular, an NGS technique emerged from bacterial CRISPR-associated protein-9 nuclease (Cas9) as a revolutionary method to modify, regulate, or mark specific genomic sequences on virtually any organism. A later adaptation of this bacterial defense mechanism that successfully and permanently edits dysfunctional genes and corrects missing proteins has resulted in a new era for disease genetic engineering. Clinical trials using this technique are already being performed, and the applicability of CRISPR-Cas9 techniques is actively being investigated using in vivo studies. However, the concept of genome correction poses great concerns from a regulatory perspective, especially in terms of security, so principles for the regulation of these methodologies are being established. We delved into CRISPR-Cas9 from its natural and ortholog origins to its engineered variants and behaviors to present its notable and diverse applications in the fields of biotechnology and human therapeutics.Entities:
Keywords: CRISPR-Cas9; NGS; bacterial adaptive defense system; clinical trials; genetic engineering
Mesh:
Substances:
Year: 2018 PMID: 29714581 DOI: 10.1177/2168479018762798
Source DB: PubMed Journal: Ther Innov Regul Sci ISSN: 2168-4790 Impact factor: 1.778