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Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
Published on: May 16, 2025
239
Direct Convective Delivery for Nervous System Gene Therapy.
James Bradley Elder1, Russell R Lonser1
1Department of Neurological Surgery, The Ohio State University Wexner Medical Center, The Ohio State University, Columbus, OH 43210, USA.
Neurosurgery Clinics of North America
|November 14, 2024
Summary
Convection-enhanced delivery offers a promising gene therapy approach for central nervous system disorders like brain tumors and epilepsy. Clinical trials are exploring its benefits and challenges for treating neurological conditions.
Area of Science:
- Neuroscience
- Gene Therapy
- Biotechnology
Background:
- Central nervous system (CNS) gene therapy is an emerging strategy.
- Neurologic conditions often have limited treatment options.
Purpose of the Study:
- To review convection-enhanced delivery (CED) for CNS gene therapy.
- To highlight the advantages and challenges of this therapeutic approach.
Main Methods:
- Review of ongoing clinical trials for CNS gene therapy.
- Analysis of CED as a delivery method.
Main Results:
- CED is a viable strategy for CNS gene therapy.
- Clinical trials demonstrate potential benefits.
- Challenges in implementation and efficacy exist.
Conclusions:
- Convection-enhanced delivery is a key strategy for CNS gene therapy.
- Further research is needed to overcome challenges and optimize treatment for various neurologic disorders.
