CRISPR-BASED GENOME EDITING AND ADVANCED DELIVERY SYSTEMS: RECENT INNOVATIONS, THERAPEUTIC APPLICATIONS, AND CHALLENGES IN CLINICAL TRANSLATION

Kajal; Aswathi K Biju; Ruchira Dubey; AkechJ. Deng

Kajal Research scholar, Laureate Institute of Pharmacy, Kathog, Jawalamukhi, Himachal Pradesh 176031, India
Aswathi K Biju Department of Bioinformatics, Saveetha Medical college and hospital, Saveetha Institute of Medical and Technical sciences, chennai-602105
Ruchira Dubey PhD Scholar, Department of Biochemistry, NIMS University, Jaipur, India
AkechJ. Deng Psychologist, James Lind Institute

Abstract

CRISPR-based genome editing has emerged as a promising advancement in molecular medicine, offering new opportunities for precise genetic modification and therapeutic development. This narrative review examines recent innovations in CRISPR technologies, advanced delivery systems, therapeutic applications, and challenges associated with clinical translation. Conventional CRISPR-Cas9 systems, base editing, and prime editing are discussed, emphasizing improvements in editing precision, efficiency, and versatility. Advanced delivery strategies, including viral vectors, lipid nanoparticles, polymeric carriers, and engineered virus-like particles, are evaluated for their potential to improve cellular uptake and tissue-specific targeting. Recent therapeutic developments demonstrate the potential of CRISPR-based approaches in treating inherited genetic disorders, cardiovascular and metabolic diseases, cancers, and selected infectious and neurological conditions. Clinical advances in sickle cell disease, β-thalassemia, and transthyretin amyloidosis highlight the growing importance of genome editing in precision medicine. However, off-target modifications, genomic instability, immunogenicity, delivery limitations, manufacturing complexity, and regulatory concerns remain significant barriers to widespread clinical implementation. Emerging innovations involving artificial intelligence, high-fidelity nucleases, optimized prime editors, and personalized delivery platforms may improve therapeutic accuracy and clinical applicability. Continued research, comprehensive safety evaluation, and standardized regulatory frameworks are essential for translating CRISPR-based technologies into effective, accessible, and sustainable treatments. These developments may further expand personalized therapeutic options while supporting responsible clinical adoption across diverse healthcare settings.

Author Biography

Kajal, Research scholar, Laureate Institute of Pharmacy, Kathog, Jawalamukhi, Himachal Pradesh 176031, India