Cell and gene therapeutic approaches are among the most effective methods for treating several diseases. Nucleic acid-based molecular detection methods have revolutionized viral detection, offering several advantages such as sensitivity, specificity, and speed. Direct visualization and biodistribution of the viral vector and the transgene in human cells or other model organisms by RNA in situ hybridization (ISH) is a powerful tool to establish the etiology and pathogenesis of the disease.

The webinar will discuss why RNAscope™ and BaseScope™ technology have become the industry standard for detecting RNA in situ in cells. We will discuss approaches to detect adeno-associated viruses (AAV) vector biodistribution, cellular tropism, transduction efficiency, and transgene expression in preclinical animal models. Our assay combines the molecular sensitivity of qPCR with single-cell and single-molecule sensitivity in the context of tissue morphology. BaseScope enables specific detection of single nucleotide differences and can differentiate AAV human transgene in non-human primates. For oligonucleotide therapies, you will learn how the miRNAscope™ assay can detect siRNA/ antisense oligos (ASOs) and how the RNAscope™ Plus assay can detect both the therapeutic siRNA/ ASO and up to three mRNAs including the target mRNA, simultaneously. You will also learn how a leading biopharma employed RNAscope to answer questions related to CAR T-cell therapies like on-target/on-tumor specificity, tumor infiltration, and activation. You can learn more on Professional Assay Service offerings and how it can be leveraged to detect AAVs, siRNA, and ASOs to target disease pathologies.

Learning Objectives:

• Characterize AAV vector biodistribution, cellular tropism, and transgene mRNA expression analysis.
• Quantify expression and persistence of vector transgenes including the gene-editing cargo.
  
• Co-detection of siRNA and up to three target mRNAs using RNAscope Plus assay.
• Localize CAR T cells to study on-target/on-tumor effects, tumor infiltration, and activation.