Neuron-restrictive silencer elements mediate neuron specificity of adenoviral gene expression

摘要: Neuron–restrictive silencer elements (NRSEs) were used to target the gene expression of adenoviral vectors specifically neuron cells in central nervous system. By generating constructs which NRSE sequences placed upstream from ubiquitous phosphoglycerate kinase promoter, specificity a luciferase reporter was tested both cell lines and primary cultures. Whereas transgene negligible nonneuronal following infection with an adenovirus containing 12 NRSEs, neuronal strongly expressed when infected same adenovirus. The NRSEs restricted vivo adenoviruses injected intramuscularly into mice intracerebrally rats. This strategy may avoid side effects resulting ectopic therapeutic genes treatment neurological diseases. In particular, it allow direct transfection motor neurons without promoting within inoculated muscles or secretion products bloodstream.