ADVANCEMENTS IN CRISPR-CAS9 TECHNOLOGY: IMPLICATIONS FOR GENE EDITING AND THERAPEUTIC APPLICATIONS

Abstract

Gene editing brought about by the CRISPR-Cas9 technology has transformed such an area by introducing the ability to alter genomes by relatively accurate, fast, and affordable methods.  The aims of the review are to cover all the new developments in CRISPR-based systems, including the emergence of Cas12, Cas13, base editing and prime editing technologies. Each of them has improved the target specificity tremendously and the off-target effects considerably too.  We investigated editing efficiency scores of various Cas variants (Cas9, Cas12 and Cas13), and examined over 20 clinical trials that occurred within (2020-2024) and addressed genetic diseases such as sickle cell anemia, muscular dystrophy, and cystic fibrosis.  The findings indicate that Cas12 beats Cas9 in editing and primary editing is the most mistake-free of all the platforms analyzed.  The tables and data indicate that there is an increased use of CRISPR clinical applications. The largest therapeutic application comprises of gene therapy and cancer immunotherapy.  A market projection shows that CRISPR therapies will ceramic rapidly until 2030, as because of success over the field of immuno-oncology and re- generation medicine.  Despite such advances, the matter of altering the germline, human enhancement, inequitable access, and the many ethical concerns remain.  It makes it even more difficult to apply CRISPR technologies in clinical practice because they differ in the regulatory systems of countries, and people do not quite understand what can be done with them.  In this review, CRISPR-Cas9 technologies can have potential to transform the treatment of hereditary and acquired diseases, although they require global ethical standards, safety, and equitable access systems to safely implement them.