An siRNA composition based on engineered exosomes and applications thereof

Abstract

This invention relates to a gene therapy drug for treating neurosenescence and neurodegenerative diseases, and its preparation method. The drug comprises engineered exosomes carrying small interfering RNA targeting receptor-interacting protein kinase 3 (RIPK3), possessing highly efficient delivery capabilities across the blood-brain barrier (BBB) ​​and targeting neurons. By delivering the engineered exosomes to the patient's brain, specific silencing of the RIPK3 gene is achieved. The exosome-delivered siRNA therapeutic strategy provided by this invention has the advantages of strong targeting, good biocompatibility, and high safety, offering a new and effective technical solution for the treatment of neurosenescence and neurodegenerative diseases.

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