Tissue Biodistribution of AAV-based gene therapy with the RNAscope™ assay

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 report we demonstrated the use of the RNAscope® assay to determine therapeutic AAV vector cell tropism and transgene expression in non-human primate retina.

We demonstrate the ability of the RNAscope® assay to:

• Visualize AAV vector tissue distribution and transgene expression in retina
• Simultaneously detect vector DNA and transgene RNA • Identify specific cell populations with a multiplex assay using cell markers and AAV vector probes