Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery.

ABSTRACT: Neurological diseases and disorders (NDDs) present a significant societal burden and currently available drug- and biological-based therapeutic strategies have proven inadequate to alleviate it. Gene therapy is a suitable alternative to treat NDDs compared to conventional systems since it can be tailored to specifically alter select gene expression, reverse disease phenotype and restore normal function. The scope of gene therapy has broadened over the years with the advent of RNA interference and genome editing technologies. Consequently, encouraging results from central nervous system (CNS)-targeted gene delivery studies have led to their transition from preclinical to clinical trials. As we shift to an exciting gene therapy era, a retrospective of available literature on CNS-associated g

SUBMITTER: Joshi CR 

PROVIDER: S-EPMC5393046 | biostudies-literature | 2017 Mar

REPOSITORIES: biostudies-literature