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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) has proven to be an effective delivery vehicle for gene therapy, but it is essential to understand the biodistribution of AAV in target and non-target tissues, as well as the persistence of transgene expression in those tissues.

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

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