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Non-Viral Nucleic Acid Delivery Strategies to the Central Nervous System
James-Kevin Y Tan1, Drew L Sellers1, Binhan Pham1
1Department of Bioengineering and Molecular Engineering & Sciences Institute, University of Washington Seattle, WA, USA.
Frontiers in Molecular Neuroscience
|November 17, 2016
Summary
Non-viral nucleic acid therapies show promise for central nervous system (CNS) disorders. This review details in vivo delivery challenges and strategies for non-viral vectors in CNS gene therapy.
Area of Science:
- Neuroscience
- Biotechnology
- Gene Therapy
Background:
- Central nervous system (CNS) injuries and neurological disorders are increasingly prevalent.
- Nucleic acid therapies offer potential for neuronal regeneration and halting disease progression.
- Non-viral vectors are emerging as safer alternatives to viral vectors for gene delivery.
Approach:
- This review focuses on in vivo challenges for non-viral nucleic acid delivery to the CNS.
- It explores strategies and vehicles designed to overcome these delivery barriers.
- Various administration routes, including systemic, cerebrospinal fluid, intraparenchymal, and peripheral injections, are evaluated.
Key Points:
- Non-viral vectors present fewer safety concerns and can deliver diverse nucleic acids.
- Overcoming in vivo delivery barriers is crucial for clinical translation.
- Successful non-viral gene transfer to the CNS has been demonstrated with specific vehicles and strategies.
Conclusions:
- The presented approaches can guide the development of synthetic gene delivery vectors for CNS applications.
- Advancements in non-viral vector technology are bringing them closer to clinical use.
- Effective non-viral delivery is key to unlocking the therapeutic potential of nucleic acid therapies for neurological conditions.
