Tissue biodistribution of AAV-based gene therapy

AAV tissue biodistribution and cell tropism are essential pieces of information for every gene therapy development program. The RNAscope® ISH assay is a highly informative method for quantitative, cell-specific measurement of AAV vector and transgene expression. The RNAscope® assay can provide single-cell biodistribution data for every tissue type. In this application note, ACD demonstrates the use of the RNAscope® assay to determine therapeutic AAV vector cell tropism and transgene expression in non-human primate retina.