As the therapeutic potential of various RNA species is becoming more and more apparent, there is increasing need for carrier systems that efficiently transport RNA to its target site. This project focuses on an intracellular target of RNA delivery, the mitochondria. Delivery of intact tRNAs to mitochondria would be a therapeutic approach for diseases like the MELAS syndrome that result from mutated mitochondrial tRNAs.
Studies with siRNA as model payload have already characterized liposomes as suitable systems for cellular delivery. In the next step, liposomes will be loaded with tRNAs and functionalized by incorporation of mitochondriotropic compounds into the lipid bilayer. The resulting formulations will be checked for their ability to act as carriers for mitochondrial targeting.