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Tissue Biodistribution of AAV-Based Gene Therapy with the RNAscope Assay
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"Tissue Biodistribution of AAV-Based Gene Therapy with the RNAscope Assay"

Adeno-associated virus (AAV) tissue biodistribution and cell tropism are essential pieces of information for every gene therapy development program. The RNAscope in situ hybridization assay is a 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.

This report from ACD demonstrates the use of the RNAscope assay to visualize therapeutic AAV vector cell tropism and transgene expression in non-human primate retina, simultaneously detecting vector DNA and transgene RNA and identifying specific cell populations using cell markers and vector probes.

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