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Ther Innov Regul Sci ; 52(6): 701-707, 2018 11.
Artículo en Inglés | MEDLINE | ID: mdl-29714581

RESUMEN

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.


Asunto(s)
Proteína 9 Asociada a CRISPR/metabolismo , Ingeniería Genética/legislación & jurisprudencia , Proteínas Bacterianas/metabolismo , Sistemas CRISPR-Cas , Ensayos Clínicos como Asunto/legislación & jurisprudencia , Ingeniería Genética/métodos , Genoma Humano , Secuenciación de Nucleótidos de Alto Rendimiento , Humanos , Mutación , Análisis de Secuencia de ADN
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