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Updated: Aug 14, 2026
Intracerebroventricular Viral Injection of the Neonatal Mouse Brain for Persistent and Widespread Neuronal Transduction
Published on: September 15, 2014
Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional
1Department of Neurology, University of Göttingen, Medical School S2-Laboratory, Waldweg 33, 37073, Göttingen, Germany. sebastian.kuegler@med.uni-goettingen.de
Abstract:
Adeno-associated- (AAV) based vectors are promising tools for gene therapy applications in several organs, including the brain, but are limited by their small genome size. Two short promoters, the human synapsin 1 gene promoter (hSYN) and the murine cytomegalovirus immediate early promoter (mCMV), were evaluated in bicistronic AAV-2 vectors for their expression profiles in cultured primary brain cells and in the rat brain. Whereas transgene expression from the hSYN promoter was exclusively neuronal, the murine CMV promoter targeted expression mainly to astrocytes in vitro and showed weak transgene expression in vivo in retinal and cortical neurons, but strong expression in thalamic neurons. We propose that neuron specific transgene expression in combination with enhanced transgene capacity will further substantially improve AAV based vector technology.
