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Summary

RNA-based medicines are an innovative type of treatment that uses genetic instructions to help cells produce beneficial proteins or silence harmful genes to treat diseases. This research focuses on improving how RNA-based medicines are delivered to different parts of the body using nanosized, fat-based particles called lipid nanoparticles (LNPs). Currently, most RNA therapies reach the liver upon systemic administration, but this thesis explores ways to direct them to other organs, such as tumors and the heart.

One approach tested in this study is blocking certain immune cells that naturally clear foreign particles from the body. Another method involves adding special targeting molecules to the surface of LNPs to help them find and enter specific target cells, such as prostate cancer cells. This research also investigates a local injection technique to deliver LNPs into the heart for treating heart diseases. Furthermore, it examines how well these methods work in different animal models and looks at possible treatment side effects, such as inflammation or toxicity. Additionally, this study explores the potential of extracellular vesicles—natural carriers in our bodies—as an alternative to synthetic lipid-based particles for drug delivery. By improving RNA delivery, this research could help develop better treatments for diseases like cancer and heart conditions, making therapies more effective and safer for patients.