EXOME ENGINEERING IN PRECISION MEDICINE: CRISPR-BASED THERAPEUTIC STRATEGIES FOR GENETIC DISEASES AND CANCER

Abstract

Exome engineering represents an emerging genome editing strategy that focuses specifically on protein coding regions of the genome where the majority of disease associated mutations occur. Targeted modification of these regions provides an opportunity to directly correct pathogenic variants responsible for inherited disorders and cancer. Recent advances in genome editing platforms, particularly CRISPR/Cas9, base editing, and prime editing technologies, have significantly improved the precision and efficiency of gene modification. Parallel progress in delivery systems has also accelerated the translation of gene editing into clinical applications. Among these systems, exosomes have attracted increasing attention as natural nanocarriers capable of transporting gene editing components across biological barriers while minimizing immune responses. In oncology, exome level analysis has facilitated the identification of tumor specific mutations and neoantigens, enabling more precise therapeutic approaches such as adoptive cell therapy and personalized immunotherapies. In particular, cancers such as oral squamous cell carcinoma may benefit from targeted editing of driver mutations including TP53, KRAS, and EGFR. Despite these advances, challenges related to delivery efficiency, off target effects, scalability, and regulatory oversight remain significant barriers to clinical translation. Continued developments in artificial intelligence assisted genomic analysis and improved delivery technologies are expected to accelerate progress in this field. This review uniquely integrates exome-targeted genome editing with emerging exosome-based delivery systems, providing a focused perspective on their combined therapeutic potential. Collectively, these advances position exome engineering as a promising and evolving strategy in precision medicine with significant clinical potential.