RNAi Therapeutic Delivery by Exosomes

摘要: Since the discovery of short interfering RNAs (siRNAs), their potential as a therapeutic platform has been widely recognized. However, clinical translation stalled by inefficient delivery in vivo. While some success achieved with cationic lipids and lipid-like materials for RNAi to liver, across blood–brain barrier (BBB) central nervous system treatment neurological disorders such Parkinson’s, Alzheimer’s, Huntington’s disease remains challenge. To address problem BBB, our laboratory exploited one nature’s mechanisms intercellular communication, named exosomes. They are class membrane vesicles derived from endolysosomal compartment implicated cell–cell communication shuttling various proteins, lipids, between cells. We have developed method target exosomes brain-specific peptides subsequently load them siRNA targeted brain. This chapter aims at providing an insight into vesicle-mediated RNA how these vectors can be utilized therapy.