2021 Aug 5 - PAS Webinar: RNAscope™ ISH Services for Gene Therapy Spatial Biodistribution Assessment
Ruby Hsu, Ph.D.,
Director, Business Development, Advanced Cell Diagnostics
Watch Video:

RNAscope™ in situ Hybridization Services for Gene Therapy Spatial Biodistribution Assessment

Visual detection and quantification of AAV, lentiviral, and oncolytic viral vector genome biodistribution and transgene expression in tissues with RNAscope and BaseScope™ in situ hybridization. RNAscope in situ hybridization (ISH) has been published as a means of “morphology-based AAV biodistribution analysis.” 

RNAscope in situ assays of vector genome DNA and transgene mRNA combine the molecular sensitivity of qPCR with single-cell resolution and the context of tissue morphology. Multiplexing enables vector genome and transgene mRNA to be visually assessed and quantified simultaneously in a single slide. With the addition of cell marker probes in multiplex fluorescent RNAscope assays, viral tropism can be measured in any cell population. 
Specific measurement of therapeutic human transgene expression in animal models is often impossible by IHC because of the high levels of homology between transgene and animal ortholog transcripts. BaseScope ISH is another powerful tool that enables specific detection of even single nucleotide differences and can easily differentiate human transgenes from non-human primate and other animal ortholog sequences. 
These assays are enabling more informative and efficient preclinical gene therapy programs and contributing critical data in IND-enabling studies. Please join us to hear how our services are contributing to and advancing gene therapy programs worldwide.

Learning Objectives:
Key features of RNAscope gene therapy assays:
  • Monitor AAV vector biodistribution and transgene mRNA expression in conventional FFPE tissues
  • Quantitative, single-cell measurement of vector genome and transgene multiplexed with cell-type markers
  • Use off-the-shelf probes for WPRE or promoter sequences, or design custom probes in 2 weeks
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