Therapeutic potential of RNA-enriched extracellular vesicles : the next generation in RNA delivery via biogenic nanoparticles

The quest for innovative therapeutic approaches has led scientists to explore the remarkable world of extracellular vesicles known as exosomes. These tiny membrane-bound particles, ranging from 50 to 150 nanometers in size, have emerged as promising vehicles for therapeutic applications and drug delivery. Released by actively dividing cells, exosomes possess the unique ability to ferry various effector molecules, including proteins, RNA, and even DNA, from donor cells to recipient cells, thereby modulating cellular responses and offering new avenues for medical intervention.


Exploring RNA-Based Therapeutics

Among the diverse cargo encapsulated within exosomes, RNA molecules have garnered significant attention for their therapeutic potential. This includes a wide range of RNA species such as microRNAs (miRNAs), messenger RNAs (mRNAs), long non-coding RNAs (lncRNAs), and circular RNAs (circRNAs). These RNA-based therapeutics hold great promise in controlling gene expression and treating a spectrum of medical conditions, from neurodegenerative diseases to cancer and viral infections.

Unlocking the Therapeutic Potential

One of the key advantages of utilizing exosomes as drug carriers lies in their ability to protect encapsulated RNA molecules from extracellular degradation. This protective mechanism ensures the stability and integrity of RNA cargo, enhancing its efficacy in therapeutic applications. Understanding the intricate biology of cargo loading and transfer within exosomes is pivotal in unlocking their full therapeutic potential.

Applications in Disease Treatment

Exosomes offer exciting prospects for treating various medical conditions, including neurodegenerative diseases, cancer, and viral infections. These tiny vesicles hold the promise of traversing the blood-brain barrier to deliver RNA cargo and targeting tumor cells with precision, offering a safe and effective approach to RNA-based therapy in these contexts. Furthermore, the potential for exosome-based therapies to circumvent challenges associated with synthetic lipid-based systems underscores their significance in the field of drug delivery.

As our understanding of exosome biology continues to deepen, so too does our ability to harness their therapeutic potential. By exploring the biogenesis, cargo loading mechanisms, and applications of exosomes in RNA-based therapy, researchers are paving the way for transformative advances in medicine. With ongoing research efforts focused on refining exosome-based drug delivery systems and elucidating their mechanisms of action, the future holds immense promise for leveraging these tiny vesicles as powerful tools for combating a wide range of diseases and improving patient outcomes.

Muskan M, Abeysinghe P, Cecchin R, Branscome H, Morris KV, Kashanchi F. (2024) Therapeutic potential of RNA-enriched Extracellular Vesicles 1: The next generation in RNA delivery via biogenic nanoparticles. Mol Ther [Epub ahead of print]. [abstract]

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