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"Tissue-Based Gene Therapy Biodistribution Analysis Using RNAscope In Situ Hybridization"
"Tissue-Based Gene Therapy Biodistribution Analysis Using RNAscope In Situ Hybridization"
Targeted gene therapy has shown promise for treating rare diseases, particularly those that arise from single-gene defects.
This application note from ACD demonstrates how RNAscope ISH technology and Professional Assay Services can provide morphology-based, cell-specific quantification of adeno-associated virus vector DNA and therapeutic transgene mRNA expression within intact tissue, addressing questions on biodistribution, persistence, cellular tropism, and vector promotor activity.
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