CRISPR-Based Therapeutics: Molecular Mechanisms of Gene-Targeted Drug Action

Abstract

CRISPR-based therapeutics represent a transformative advancement in molecular medicine by enabling precise, programmable modification of genomic sequences for the treatment of genetic and acquired diseases. Unlike conventional pharmacological interventions that primarily modulate protein function, CRISPR technologies directly target the underlying genetic determinants of pathology, offering the potential for durable or permanent therapeutic effects. This paper examines the molecular mechanisms of CRISPR-mediated gene-targeted drug action, focusing on DNA recognition, cleavage, repair pathways, and emerging gene-editing modalities such as base editing and prime editing. By synthesizing developments in genome engineering, delivery systems, and off-target mitigation strategies, the study evaluates the clinical promise and technical limitations of CRISPR-based interventions. The findings underscore the growing role of gene-editing therapeutics in precision medicine while highlighting ethical, regulatory, and safety considerations that accompany their translational deployment.