Advances And Applications Of Crispr-cas Systems In Precision Genome Editing And Genetic Disease Therapy

Abstract

CRISPR-Cas has transformed the field of gene therapy by providing an unprecedented degree of precision and efficacy with regard to genomic modification over the last decade. These engineered platforms, originally adapted to the adaptive immune system of bacteria, use guide RNA that with the Cas protein can be used to target a particular DNA sequence, which makes it possible to repair mutations, or eliminate diseased genes or insert sequences to cure diseases. Considering such diversity of application, CRISPR has amassed a substantial potential to address a wide range of genetic conditions comprising both monogenetic diseases (e.g., cystic fibrosis and sickle cell anemia) and far more complex conditions (e.g., cancer and infectious diseases). The advent of CRISPR versions and the specific ability to edit the DNA, the Cas9, Cas12, and Cas13 have made the technology even more versatile, allowing disrupting RNA. By its general ability to flex, CRISPR has become a unified system in terms of treating options.  However, even with such broad therapeutic application, clinical translation to date is limited by one of two main barriers; the inefficiency of existing delivery methods and the immune reaction against Cas proteins. In the recent past, however, new delivery approaches have increasingly removed these barriers, particularly those based on nanoparticle systems, and optimization of high-fidelity Cas proteins has aimed at improving specificity and safety. In this regard, the field will realize greater efficiencies and effectiveness in employment of this revolutionized technology.  This review outlines the mechanisms bases of CRISPR-Cas systems, observes the use of CRISPR-Cas in medical areas in genetic diseases, discusses the current situation of clinical trials progress, and tackles the associated obstacles and ethical issues. It takes into account the current attempts to improve specificity, efficiency, and accessibility as well, highlighting the potential that Crispr has in the development of personalized medicine.