Adeno-associated Virus-mediated, Mifepristone-regulated Transgene Expression in the Brain.

ABSTRACT: Gene therapy, in its current configuration, is irreversible and does not allow control over transgene expression in case of side effects. Only few regulated vector systems are available, and none of these has reached clinical applicability yet. The mifepristone (Mfp)-regulated Gene Switch (GS) system is characterized by promising features such as being composed of mainly human components and an approved small-molecule drug as an inducer. However, it has not yet been evaluated in adeno-associated virus (AAV) vectors, neither has it been tested for applicability in viral vectors in the central nervous system (CNS). Here, we demonstrate that the GS system can be used successfully in AAV vectors in the brain, and that short-term induced glial cell line-derived neurotrophic factor (GDNF) expres

SUBMITTER: Maddalena A 

PROVIDER: S-EPMC3731885 | biostudies-literature | 2013 Jul

REPOSITORIES: biostudies-literature