CLINICALLY SAFE THERAPEUTIC DELIVERY USING HUMAN MESENCHYMAL STEM/STROMAL CELL-DERIVED EXOSOMAL HYBRIDS

Abstract

As gene therapy techniques become more aggressive with site-specific therapeutics aimed at treating diseases such as hereditary, infectious, and neoplastic diseases at the DNA level, the parallel development of standardized, clinically safe delivery methods is paramount. Completion of this study will result in a clinically safe method to deliver large therapeutic payloads in vitro, while avoiding randomly integrated sites within the genome, incomplete therapeutic effects, and presentation of other unwanted activity typically attributed to other common viral-based delivery methods. The proposed exosome-liposome hybrid will serve as a baseline for the systems’ translational potential as further modifications to the liposomal construct must be explored before clinical application.