First extracellular vesicles (EVs) as therapeutic vehicles targeting the brain containing engineered miRNA-based silencing sequences to treat Machado-Joseph Disease (MJD) upon daily intranasal administration.

Advantages:
Packaging cell line to continuously produce engineered EVs
5x more delivery due to a specific tropism for neurons
2.5-fold enrichment of silencing sequences into EVs due to the presence of ExoMotifs
Non-invasive administration: Intranasal route

An alternative and efficient delivery method for miRNAs/silencing sequences to treat MJD and other brain diseases