Exosomes in brain diseases: Mechanisms, advances, and perspectives

Area of Science:

• Neuroscience
• Biotechnology
• Drug Delivery

Background:

• The blood-brain barrier (BBB) severely limits drug delivery for central nervous system (CNS) disorders.
• Conventional therapies face challenges with brain penetration, specificity, and side effects.
• Exosomes, as natural vesicles, offer biocompatibility and potential for BBB interaction.

Purpose of the Study:

• To review exosome-based strategies for CNS therapeutic delivery.
• To explore exosome-BBB interactions and transport mechanisms.
• To analyze exosome engineering for enhanced therapeutic performance and clinical translation.

Main Methods:

• Summarizing BBB structure and exosome transport mechanisms.
• Discussing exosome isolation, characterization, and engineering techniques.
• Analyzing exosome applications in neurodegenerative diseases, brain tumors, and ischemic stroke.

Main Results:

• Exosomes demonstrate potential for overcoming BBB limitations in CNS drug delivery.
• Engineering strategies can optimize exosome cargo, targeting, and function for specific CNS disorders.
• Pathology-specific designs are crucial for effective exosome-based CNS therapies.

Conclusions:

• Exosomes represent a promising platform for CNS therapeutics, with engineering crucial for efficacy.
• Addressing manufacturing, pharmacokinetics, safety, and regulatory hurdles is vital for clinical translation.
• A pathology-driven, engineering-guided approach is essential for developing exosome-based CNS treatments.