Designer exosomes produced by implanted cells intracerebrally deliver therapeutic cargo for Parkinson’s disease treatment

Ryosuke Kojima, Daniel Bojar, Giorgio Rizzi, Ghislaine Charpin-El Hamri, Marie Daoud El-Baba, Pratik Saxena, Simon Ausländer, Kelly R. Tan and Martin Fussenegger ()
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Ryosuke Kojima: Department of Biosystems Science and Engineering
Daniel Bojar: Department of Biosystems Science and Engineering
Giorgio Rizzi: University of Basel
Ghislaine Charpin-El Hamri: Institut Universitaire de Technologie (IUTA)
Marie Daoud El-Baba: Institut Universitaire de Technologie (IUTA)
Pratik Saxena: Department of Biosystems Science and Engineering
Simon Ausländer: Department of Biosystems Science and Engineering
Kelly R. Tan: University of Basel
Martin Fussenegger: Department of Biosystems Science and Engineering

Nature Communications, 2018, vol. 9, issue 1, 1-10

Abstract: Abstract Exosomes are cell-derived nanovesicles (50–150 nm), which mediate intercellular communication, and are candidate therapeutic agents. However, inefficiency of exosomal message transfer, such as mRNA, and lack of methods to create designer exosomes have hampered their development into therapeutic interventions. Here, we report a set of EXOsomal transfer into cells (EXOtic) devices that enable efficient, customizable production of designer exosomes in engineered mammalian cells. These genetically encoded devices in exosome producer cells enhance exosome production, specific mRNA packaging, and delivery of the mRNA into the cytosol of target cells, enabling efficient cell-to-cell communication without the need to concentrate exosomes. Further, engineered producer cells implanted in living mice could consistently deliver cargo mRNA to the brain. Therapeutic catalase mRNA delivery by designer exosomes attenuated neurotoxicity and neuroinflammation in in vitro and in vivo models of Parkinson’s disease, indicating the potential usefulness of the EXOtic devices for RNA delivery-based therapeutic applications.

Date: 2018
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DOI: 10.1038/s41467-018-03733-8

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