Guided by RNAscope, Powerful Outcomes in Gene Therapy
Unlock the potential of gene therapy with our cutting-edge RNAscope™ in situ hybridization (ISH) technology. Visualize and analyze effective delivery, biodistribution, and safety of the therapeutic with our highly sensitive solution. Quantify changes in gene expression and cellular function confidently, optimizing therapeutic efficacy and safety. Gain spatial information on the biodistribution of the AAV vector and transgene mRNA in cells and tissues. Evaluate efficacy and safety of the oligonucleotide therapy by simultaneously detecting the antisense oligos (ASO) or small RNA (siRNA/miRNA) along with the target transcript and other cell markers.
Why Choose RNAscope Technology for Gene Therapy?
RNAscope offers a groundbreaking sensitive, specific and reliable approach to gene therapy. With its unique ability to visualize and quantify RNA within individual cells, RNAscope opens up a world of possibilities for precise and targeted treatments.
Visualize Biodistribution and Transgene Expression with RNAscope ISH
- Visualize tissue biodistribution and cellular tropism of viral vectors such as AAV, transgene and RNA therapeutics delivered by non-viral platforms with single-molecule sensitivity and sub-cellular resolution.
- Select engineered vectors and capsids for highest transduction efficiency.
- Quantify expression and persistence of transgenes including codon-optimized or gene-edited cargo.
- Evaluate therapeutic efficacy including RNA efficiency within targeted cells and tissues.
- Improve safety by evaluating dosing and identifying off-target events in relevant organs.
Unlock Answers to Critical Questions
Empowering Biodistribution and Safety Analysis with Powerful RNA Visualization Tools
Visualize Biodistribution
Optimize the targeting efficiency of your therapeutic using any preclinical animal model.
Easily measure biodistribution, cellular tropism and transduction efficiency across multiple tissues of interest with high specificity.
Single Cell Gene Expression
Evaluate the performance of your engineered cargo in vivo.
Quantify the expression and persistence of your transgene or therapeutic target with single-molecule sensitivity.
Functional Impact
Demonstrate the functional efficacy and safety of your therapeutic in disease models.
Visualize changes in targeted cells and tissues in spatial context using both protein and RNA biomarkers.
Monitor off target events by visualizing biodistribution of the therapy in various organs.
RNAscope Probes Redefines Biodistribution Monitoring with Unmatched Specificity
Trusted technology used in >8,000 publications with multiple ordering formats to choose from.
We offer ultimate flexibility in RNAscope probe design. Easily design probes specific to your proprietary therapeutic for use in any animal model system.
- Enable precise distinction between human transgenes and cross-species orthologs in animal models.
- Accurately differentiate between codon-optimized transgenes and native sequences.
AAV/vector-based gene therapy
Visualize Biodistribution and Transgene Expression with RNAscope ISH
ASO and siRNA gene therapy
Visualize and Quantify Small RNAs With Morphological Context and Spatial Resolution
(1) S12 Nonclinical Biodistribution Considerations for Gene Therapy Products; Guidance for Industry; U.S. Department of Health and Human Services Food and Drug Administration; Center for Drug Evaluation and Research (CDER); Center for Biologics Evaluation and Research (CBER) May 2023; ICH-Safety
CRISPR Flyer
More Informative than qPCR
Evaluate Subcellular Localization of Vector Delivery and Transgene Expression
Morphology-based biodistribution is extremely important in the field of therapies involving gene editing. While qPCR-based methods provide average values for copy number from extracted nucleic acids, the RNAscope technology provides morphology-based quantification.
Fig. RNAscope Uniquely Enables Visualization of Cellular Uptake and Cellular Tropism. (A) AAV vector is primarily sequestered in the interstitial space. (B) HALO ® (Indica Labs) overlay identifies AAV-positive nuclei in red and AAV-negative nuclei in white. (C) Quantification of % of signal detected within the nuclear boundaries vs signal located outside of the nucleus.
Products
Probes/Targets
Catalog Probes (Ex: CMV, CBA, WPRE, etc.)
If your gene of interest in not listed in our catalog, ACD can design and manufacture new in situ hybridization probes for you, To order or get more info on Made-to-Order New Targets Probes, please fill in the form below and an ACD Account Executive will contact you.
Made-to-OrderProfessional Assay Services
- Unparalleled expertise in RNAscope and BaseScope ISH
- High capacity and high throughput, delivering more than 10,000 slides per year
- Supporting dozens of gene therapy companies for research studies enabling pre-IND submission
- Team of specialists perform quantitative image analysis using HALO® Software (Indica Labs)
- Board-certified pathologist review
- Actionable results delivered in weeks rather than months
Resources
Recorded Webinar
Visual detection and quantification of AAV and LV vector biodistribution and transgene expression in preclinical animal models with RNAscope and BaseScope™ in situ hybridization.
Watch Now
Publications
- Kinoshita, Y., Mohamed, F.F., Amadeu de Oliveira, F., Narisawa, S., Miyake, K., Foster, B.L. and Millán, J.L. (2021), Gene Therapy Using Adeno-Associated Virus Serotype 8 Encoding TNAP-D10 Improves the Skeletal and Dentoalveolar Phenotypes in Alpl−/− Mice. J Bone Miner Res
- Presa M, Bailey RM, Davis C et al. (2021) AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model. The Journal of clinical investigation.
- Bricker TL, Darling TL, Hassan AO et al. (2021) A single intranasal or intramuscular immunization with chimpanzee adenovirus-vectored SARS-CoV-2 vaccine protects against pneumonia in hamsters. Cell reports
- White KA, Nelvagal HR, Poole TA et al. (2021) Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice. Molecular Therapy - Methods & Clinical Development
- Zhao J, Yue Y, Patel A et al. (2020) High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection. Molecular Therapy - Methods & Clinical Development.
- Di Maria V, Moindrot M, Ryde M, Bono A, Quintino L, Ledri M.(2020) Development and Validation of CRISPR Activator Systems for Overexpression of CB1 Receptors in Neurons. Front Mol Neurosci.
Application Note
Application note "Tissue Biodistribution of AAV-based gene therapy with the RNAscope™ assay'
