Tissue biodistribution of AAV-based gene therapy

18 Jan 2022

AAV tissue biodistribution and cell tropism are essential pieces of information for every gene therapy development program. 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 application note, ACD demonstrates the use of the RNAscope® assay to determine therapeutic AAV vector cell tropism and transgene expression in non-human primate retina.

RNAscope™ Chromogenic Assays

Advanced Cell Diagnostics

Independent of your genes of interest, our easy RNA in situ hybridization assay uses common reagents and protocols; providing universal assay conditions. All RNA-ISH kits contain reagents in a convenient ready-to-use (RTU) format, and chromogenic dyes are available to interrogate a single or two genes simultaneously.

(1)

Links

Tags

Cell-Based AssaysCell-based assays are used to monitor the presence, quantity and activities of a desired cellular analyte including drug molecules or biomarkers. This can reveal information on cell health (apoptosis, cytotoxicity, viability and proliferation assays), cell metabolism, cell migration and cell signaling mechanisms. Find the best cell-based assay products, kits and equipment with our peer reviewed product directory: compare products, check customer reviews and receiving pricing direct from manufacturers.Tissue ProcessingTissue processing involves preparing biological tissue samples for microscopic analysis, including fixation, embedding, and sectioning. This process is essential for histopathology and diagnostic applications. Explore tissue processing products in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.VirusesViruses are microscopic pathogens that require a host cell to replicate. Understanding their structure, replication cycle, and impact on the immune system is crucial in developing effective treatments, vaccines, and diagnostic methods. Research continues to focus on emerging viral diseases and antiviral drug discovery. Explore solutions for virus research, detection, and treatment in our peer-reviewed product directory; compare products, check customer reviews, and get pricing directly from manufacturers.FFPEFormalin-fixed paraffin-embedded (FFPE) tissues are commonly used in medical research, particularly in histology and cancer studies. The preservation process allows long-term storage of tissue samples for later analysis. Explore FFPE research products in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.Gene TherapyGene therapy is a technique that modifies or replaces genes within an individual's cells to treat or prevent diseases. This approach holds potential for curing genetic disorders, cancers, and certain viral infections. Advances in gene editing technologies like CRISPR are driving the growth of gene therapy. Explore gene therapy tools and technologies in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.
Tissue biodistribution of AAV-based gene therapy