When Lydia Alvarez-Erviti started her postdoctoral studies at the University of Oxford, UK, in 2008, her goal was to develop gene therapies for neurodegenerative diseases. She had identified her target — α-synuclein, a protein that accumulates in the brains of people with Parkinson’s disease — and designed a short interfering RNA (siRNA) to reduce the amount of α-synuclein made in mice. But she needed to get the siRNA into the brain. The method would have to protect the RNA, cross the barrier between circulating blood and the brain, and be safe enough to use repeatedly. Fortuitously, a colleague had begun studying something that could work — naturally occurring, nano-sized vesicles called exosomes.
Around ten years ago, Alvarez-Erviti, who is now at the Center for Biomedical Research of La Rioja, Spain, and her colleagues proved exosomes’ potential as drug carriers in a mouse model of Parkinson’s disease. Now, a large body of a work in animals, along with early studies in people, has demonstrated the proficiency and safety of exosome products.

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