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

SUBMITTER: Puhl DL 

PROVIDER: S-EPMC8284997 | biostudies-literature | 2019 Aug

REPOSITORIES: biostudies-literature