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Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
Seigo Kimura1,2, Hideyoshi Harashima1,2
1Laboratory for Molecular Design of Pharmaceutics, Faculty of Pharmaceutical Sciences, Hokkaido University, Sapporo 060-0812, Japan.
Pharmaceutics
|December 18, 2020
Summary
Gene therapy offers promise for neurological disorders like Alzheimer's and Parkinson's. Nanotechnology-based delivery systems are key to overcoming the blood-brain barrier challenge for effective central nervous system treatment.
Area of Science:
- Neurology
- Biotechnology
- Nanomedicine
Background:
- Aging populations are increasing the prevalence of neurological disorders such as Alzheimer's disease (AD) and Parkinson's disease (PD).
- These conditions pose significant societal and economic burdens globally.
- Current gene therapy options for central nervous system (CNS) disorders are limited, with drug delivery to the brain being a major obstacle.
Purpose of the Study:
- To comprehensively review the current status of drug delivery systems for CNS disorders, focusing on gene therapy.
- To explore advancements in overcoming the blood-brain barrier (BBB) for neurological treatments.
- To highlight the role of nanotechnology in developing targeted gene delivery.
Main Methods:
- Review of existing literature on CNS drug delivery and gene therapy.
- Analysis of blood-brain barrier (BBB) functions and strategies to overcome it.
- Evaluation of viral vectors, specifically adeno-associated viral (AAV) vectors, for gene delivery.
- Assessment of non-viral, non-invasive methods for brain targeting.
Main Results:
- Nanotechnology, utilizing viral and non-viral vectors, enables efficient brain-targeted gene delivery systems.
- Adeno-associated viral (AAV) vectors represent a leading technology in gene delivery for CNS applications.
- Non-invasive, non-viral methods show potential for brain targeting.
Conclusions:
- Targeted gene delivery using nanotechnologies is crucial for advancing the treatment of neurological disorders.
- Overcoming the blood-brain barrier remains a critical challenge, with AAV vectors and non-viral methods offering promising solutions.
- Further development in these areas is essential to realize the full potential of gene therapy for CNS diseases.
