THE APPLICATION OF CRISPR TECHNOLOGY IN TRANSLATIONAL RESEARCH: CURRENT CHALLENGES AND FUTURE DIRECTIONS

Abstract

CRISPR-Cas9 technology has revolutionized the landscape of genetic engineering by enabling highly precise, efficient, and programmable genome editing. Originally derived from bacterial immune systems, CRISPR has emerged as a pivotal tool in translational research with broad applications in genetic therapies, disease modeling, and agricultural biotechnology. Despite its transformative potential, real-world deployment of CRISPR faces significant scientific, regulatory, and ethical barriers.This study systematically evaluates the clinical and agricultural applications of CRISPR, employing a mixed-methods approach that includes gene-editing efficiency analyses, delivery system optimization, and off-target profiling. Mechanistic investigations were also conducted to compare editing precision across Cas9 and next-generation systems such as Cas12 and Cas13.The results demonstrate that CRISPR-Cas9 achieves over 85% target-specific efficiency across diverse gene targets, with significantly reduced off-target effects using high-fidelity variants. Delivery success rates vary across vector types, with lipid nanoparticles and electroporation outperforming viral vectors in safety and specificity. In agricultural models, the technology enhanced crop resilience and nutrient content while maintaining genetic stability across generations. Further, editing success was highest in ex vivo somatic cell contexts, highlighting its near-term feasibility in personalized medicine.These findings underscore CRISPR’s readiness for controlled deployment in clinical and agricultural settings, particularly when integrated with precision delivery and validation protocols. Nonetheless, the study highlights unresolved issues including the ethical implications of germline editing, public trust, and unequal access to gene therapies. As CRISPR advances toward routine therapeutic and agricultural use, addressing regulatory harmonization, biosafety, and equity concerns will be essential. This research provides a foundational assessment of the technology’s current capabilities and outlines future directions for responsible integration into translational practice.