World Journal of
Pharmaceutical and Life Sciences

ADVANCES AND APPLICATIONS OF CRISPR/Cas9 TECHNOLOGY IN GENE EDITION

Alicia Armentia, María San Miguel, Angel San Miguel*, Sara Martín, Blanca Martín, Jose Antonio Garrote

ABSTRACT

CRISPR/Cas9 technology has transformed the field of gene editing by providing a precise, efficient, and versatile tool to modify DNA in a wide variety of organisms. This system, based on the immune system of bacteria and archaea, consists of a guide RNA molecule and the Cas9 enzyme. The guide RNA is designed to recognize specific DNA sequences, and when it binds to its target, the Cas9 enzyme cuts the DNA at that site. From there, the natural cellular repair mechanisms come into play and can result in the insertion or deletion of bases or the introduction of a new DNA sequence. The technology has found numerous applications in various fields. In medicine, it is being used for research into genetic diseases, allowing the study of the molecular bases of various conditions and the development of gene therapies. The ability to edit genes in human cells and tissues opens up the possibility of treating inherited and acquired diseases. In addition, CRISPR/Cas9 has facilitated the creation of animal models to study diseases and evaluate new therapies. In the field of agriculture, it is being used to develop crops resistant to diseases, pests or adverse environmental conditions. Research is also being carried out to improve the nutritional quality of crops, increase agricultural productivity and reduce the use of agrochemicals. These applications can contribute to addressing global food and food security challenges. In biotechnology, the technology has great potential for the production of pharmaceuticals and enzymes. It allows the precise modification of microorganisms to increase the efficiency of the production of biochemical and pharmaceutical substances of interest. Likewise, CRISPR/Cas9 is used in the engineering of microorganisms for industrial applications, such as the production of bioplastics or biofuels. Recently, significant advances have been made in CRISPR/Cas9 technology to improve its efficiency and accuracy. Additional variants of CRISPR systems, such as Cas13 and Cas12, are being developed that extend the gene editing capabilities and address certain challenges and limitations present in the original Cas9 system. In addition, approaches are being investigated to reduce potential side effects and improve the specificity of gene editing. While this technology has fueled important advances, it also poses security and ethical challenges. The debate on gene editing in human embryos and the long-term implications of modifying the human genome continues. It is critical to ensure responsible and ethical use of technology, as well as address potential risks and legal considerations associated with gene editing. As this tool continues to be researched and refined, it is essential to carefully assess its safety and ethical implications, while promoting its responsible application for the benefit of society.

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