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eExos-miR-124 is an engineered exosome-based gene delivery tool designed for the treatment of glioblastoma. These exosomes (eExos) are modified to express retroviral Gag and VSVg proteins, which enhances the packaging efficiency of genetic cargo and facilitates cellular entry. The therapeutic payload is microRNA-124 (miR-124), a potent anti-glioma microRNA identified through high-throughput screening for its ability to inhibit the proliferation of glioma stem cells (GSCs). In preclinical models, eExos-miR-124 has demonstrated the ability to deliver miR-124 to brain tumor cells, leading to decreased tumor cell proliferation and improved survival in mouse models of glioma. The platform aims to overcome delivery barriers in the central nervous system by utilizing the natural trafficking properties of exosomes combined with viral protein-mediated enhancement.
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