Abstract

CRISPR TECHNOLOGY AND ITS TRANSFORMATIVE ROLE IN MODERN PHARMACOLOGY

Bachu Bhavani*, Chandramouli Sunkara, R.L. Manisha, Sudhakar Muvvala

ABSTRACT

This report provides a comprehensive analysis of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology and its transformative impact on modern pharmacology. Originating as a bacterial adaptive immune system, CRISPR-Cas has been repurposed into a revolutionary gene-editing tool, distinguished by its precision, efficiency, and programmability. The report details the historical discovery, fundamental mechanism of action, and the diversification of CRISPR-Cas systems, including Cas9, Cas12, and Cas13, each offering unique capabilities for targeting DNA and RNA. A significant portion of the analysis is dedicated to CRISPR's broad applications in biological and medical research, such as precise gene editing and regulation, the creation of high-fidelity cellular and animal disease models, and its role in functional genomics. The document further explores CRISPR's pivotal function in the drug discovery and development pipeline, where it has streamlined target identification and validation, provided novel strategies to understand and overcomedrug resistance, and enhanced high-throughput screening processes. The report also examines the direct therapeutic applications of CRISPR, highlighting its clinical success in treating genetic disorders like sickle cell anemia and its role in engineering the next generation of cancer immunotherapies, such as CAR T-cells. Despite its promise, the technology faces significant challenges, including the risk of off-target effects and the complexities of safe and efficient in vivo delivery, which are discussed in detail. Finally, the document addresses the profound ethical considerations surrounding CRISPR, with a particular focus on the distinction between somatic and germline editing, and the broader societal implications related to safety, equity, and access. The report concludes with an outlook on the future of CRISPR in pharmacology, projecting its central role in advancing personalized medicine and precision therapies through continuous technological innovation and an evolving regulatory landscape.

Keywords: CRISPR, Gene Editing, Drug Discovery, Target Identification, High-Throughput Screening (HTS), Disease Modeling, Personalized Medicine, CAR T-cell Therapy, Off-target Effects.