Challenges of Gene Delivery to the Central Nervous System and the Growing Use of Biomaterial Vectors

Abstract

Gene therapy is a promising form of treatment for those suffering from neurological disorders or central nervous system (CNS) injury, however, obstacles remain that limit its translational potential. The CNS is protected by the blood brain barrier, and this barrier blocks genes from traversing into the CNS if administered outside of the CNS. Viral and non-viral gene delivery vehicles, commonly referred to as vectors, are modified to enhance delivery efficiency to target locations in the CNS. Still, there are few gene therapy approaches approved by the FDA for CNS disease or injury treatment. The lack of viable clinical approaches is due, in part, to the unpredictable nature of many vector systems. In particular, safety concerns exist with the use of viral vectors for CNS gene delivery. To seek some alternatives to viral vectors, development of new non-viral, biomaterial vectors is occurring at a rapid rate. This review discusses the challenges of delivering various forms of genetic material to the CNS, the use and limitations of current viral vector delivery systems, and the use of non-viral, biomaterial vectors for CNS applications.

Department(s)

Chemical and Biochemical Engineering

Comments

National Science Foundation, Grant C30606GG

Keywords and Phrases

Biomaterials; Central nervous system; Gene delivery; Non-viral vectors

International Standard Serial Number (ISSN)

1873-2747; 0361-9230

Document Type

Article - Journal

Document Version

Final Version

File Type

text

Language(s)

English

Rights

© 2025 Elsevier, All rights reserved.

Publication Date

01 Aug 2019

PubMed ID

31173859

Link to Full Text

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