AAV vector-mediated cell type-specific gene knock-down / knock-out in marmoset CNS in vivo
Graduate School of Medicine,
Viral vectors carrying cell type-specific promoters allow us to express transgenes specifically in certain cell populations in a temporally regulated manner. We have developed cell type-specific promoters available in marmoset central nervous system, including astrocyte-specific, neuron-specific and cerebellar Purkinje cell-specific promoters. In the present study, we validate cell type-specific gene knock-down and knock-out in marmoset brain by combination of AAV vectors carrying the cell type-specific promoters with miRNA or CRSPR/Cas9 system and subsequently challenge generation of marmoset model of neuropsychiatric disorders using the explored vectors.