AAV9-hSyn-Luc

The recombinant adeno-associated virus serotype 9 vector (AAV9-hSyn-Luc), expressing firefly luciferase under the control of the human synapsin 1 promoter (hSyn), is a powerful and versatile tool in neuroscience research, combining several key advantages. First, AAV9 is renowned for its exceptional tropism for central nervous system (CNS) cells. Unlike many other AAV serotypes, AAV9 can efficiently cross the blood-brain barrier (BBB) ​​after systemic administration, enabling efficient and widespread transduction of neurons and glial cells in the brain and spinal cord without the need for invasive intracranial injections, although direct CNS delivery remains an option for targeted studies. Second, the human synapsin 1 (hSyn) promoter provides highly specific and robust neuronal expression. Synapsin is a protein primarily expressed in neurons, and the hSyn promoter fragment used in this vector selectively drives transgene expression in neuronal populations, minimizing off-target expression in non-neuronal cells of the CNS, thereby increasing the specificity and relevance of experimental results. Finally, the firefly luciferase (Luc) reporter gene offers a unique advantage: non-invasive, longitudinal bioluminescence imaging (BLI). After transduction, luciferase expression can be repeatedly and quantitatively monitored over time in the same animal using sensitive optical imaging systems after administration of its substrate, D-luciferin.

AAV9-hSyn-Luc has broad applications across various fields of neuroscience. One of its primary uses is in studying neuronal transduction profiles and gene expression dynamics. Researchers use this vector to map the efficiency and specificity of AAV9 (and potentially related capsids) neuronal targeting in different brain regions, developmental stages, or disease models, via various routes of administration (e.g., intravenous, intrathecal, intracerebroventricular, intraparenchymal). The longitudinal nature of BLI allows for detailed studies of how transduction and expression evolve over time (weeks or months), providing crucial data on vector dynamics. Secondly, it is also a valuable tool for developing and validating novel gene therapy strategies for neurological diseases.