期刊
BRAIN RESEARCH BULLETIN
卷 150, 期 -, 页码 216-230出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.brainresbull.2019.05.024
关键词
Biomaterials; Gene delivery; Central nervous system; Non-viral vectors
资金
- NIH R01 [NS092754]
- State of New York Spinal Cord Injury Research Program Institutional Grant [C32245GG]
- New York Spinal Cord Injury Research Board Predoctoral Fellowship Award [C30606GG]
- National Science Foundation Graduate Research Fellowship Program [DGE-1744655]
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.
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