Mifepristone-inducible transgene expression in neural progenitor cells in vitro and in vivo.

ABSTRACT: Numerous gene and cell therapy strategies are being developed for the treatment of neurodegenerative disorders. Many of these strategies use constitutive expression of therapeutic transgenic proteins, and although functional in animal models of disease, this method is less likely to provide adequate flexibility for delivering therapy to humans. Ligand-inducible gene expression systems may be more appropriate for these conditions, especially within the central nervous system (CNS). Mifepristone's ability to cross the blood-brain barrier makes it an especially attractive ligand for this purpose. We describe the production of a mifepristone-inducible vector system for regulated expression of transgenes within the CNS. Our inducible system used a lentivirus-based vector platform for the ex viv

SUBMITTER: Hjelm BE 

PROVIDER: S-EPMC4976764 | biostudies-literature | 2016 May

REPOSITORIES: biostudies-literature