Tissue Biodistribution Of AAV-Based Gene Therapy With The RNAscope® Assay

By Courtney Anderson, Nidhi Vashistha, Mansi Garg, Xiao-Jun Ma, Chris Bunker, Ruby Hsu, Advanced Cell Diagnostics Inc.

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