Exosome Nanotechnology in Molecular Medicine: Advances, Applications and Challenges in Gene Therapy

Abstract

Content of image described in text. Abstract Background Gene therapy has emerged as a transformative approach for treating diseases that are caused by genetic defects, including cancer, inherited metabolic disorders and immunodeficiencies. However, its clinical success depends critically on the availability of safe, efficient and non-immunogenic delivery systems. Conventional viral vectors, while highly effective, are associated with immunogenicity, oncogenic risk and high production costs. Non-viral alternatives offer improved safety but they have lower transfection efficiency and limited stability. Exosomes are naturally secreted, endosome-derived nanovesicles ranging from 30–150 nm in diameter and have emerged as promising next-generation carriers for gene and drug delivery. Their inherent biocompatibility, low immunogenicity, ability to cross biological barriers and capacity to transport diverse molecular cargo including nucleic acids, proteins and small molecules make them particularly attractive as therapeutic platforms. Methods This review summarizes the biogenesis, molecular composition and classification of exosomes and examines their applications in siRNA/miRNA delivery, CRISPR/Cas9 gene editing, mRNA therapy, cancer treatment and vaccine development. Special attention is given to engineered, plant-derived and stem cell-derived exosomes, as well as current cargo-loading strategies and commercial therapeutic platforms. Results Despite their promise, significant challenges remain, including low cargo-loading efficiency, batch heterogeneity, limited scalability and the absence of standardized manufacturing and regulatory frameworks. Conclusions Future research must address these barriers to accelerate the clinical translation of exosome-based therapeutics.