NUCLEIC ACID-BASED THERAPEUTIC DELIVERY SYSTEMS: GENE THERAPY PRINCIPLES, DELIVERY STRATEGIES, AND LIPOSOMAL APPROACHES: A REVIEW
B. Premkumar*, D. Dhachinamoorthi, M. Sujith
ABSTRACT
Nucleic acid therapeutics, including plasmid DNA, mRNA, siRNA, antisense oligonucleotides, and CRISPR-based systems, have revolutionized modern medicine by enabling targeted treatment of genetic and acquired diseases. Gene therapy involves the delivery of genetic material to correct, replace, silence, or supplement defective genes through ex-vivo or in-vivo approaches. It has shown significant therapeutic potential in inherited disorders such as severe combined immunodeficiency, haemophilia, spinal muscular atrophy, inherited retinal diseases, and various cancers, including CAR-T cell therapy. The success of gene therapy depends on efficient and safe delivery systems. Viral vectors provide high gene transfer efficiency but are limited by immunogenicity, insertional mutagenesis, and manufacturing challenges. Non-viral approaches, including physical methods and chemical carriers, offer improved safety with advancing delivery efficiency. Among these, liposomal systems, particularly ionizable lipid nanoparticles (LNPs), have become clinically established platforms for nucleic acid delivery, as demonstrated by the siRNA drug patisiran and mRNA COVID-19 vaccines. This review summarizes gene therapy strategies, delivery systems, liposomal gene delivery technologies, their clinical applications, and future perspectives.
Keywords: Gene Therapy, Nucleic Acid Delivery, Viral Vectors, Non-viral Vectors, Liposomal Gene Delivery, Ex-vivo Gene Therapy, In-vivo Gene Therapy, Cationic Liposomes, Lipid Nanoparticles.
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