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Visualize and Quantify | Assess Biodistribution and Function | Products & Services | Resources 

Visualize and Quantify Small RNA Delivery and Function

Synthetic oligonucleotides like small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs) function as a form of gene therapy by precisely targeting genes to control their expression. With RNAscope ISH technology you can visualize small regulatory RNAs (smRNAs) alone, or along with its mRNA targets. Assess oligonucleotide therapy delivery, biodistribution, and interrogate smRNA biomarkers along with cell specific/morphology/functional biomarkers.​

Unlock Answers to Critical Questions

Questions for Your Oligonucleotide-Based Studies

Visualize Synthetic and Endogenous RNA to​ Advance Your Small RNA Therapeutic​

  • Assess in vivo biodistribution of the ASOs or small RNA and determine cellular uptake, and persistence over time.​
  • Visualize 1 small RNA (ASO, miRNA, siRNA) + up to 3 mRNA targets in intact tissues.​
  • Evaluate expression of the target gene and cell-specific marker genes with morphological context in heterogeneous tissues. ​
  • Safety and efficacy screen for potential toxicity and off-target effects.
FDA strongly recommends biodistribution studies to characterize engineered products (1). RNAscope in situ hybridization assay is the most powerful spatial method available within the methods provided.

Assess Biodistribution and Functional Effects of Small RNA Therapeutics

miRNAscope™ Assay illustration 1

miRNAscope™ Assays

The miRNAscope assay allows visualization of ASOs, miRNA, siRNA, and other nucleic acid targets between 17-50 nucleotides.​

RNAscope PLUS Assays illustration

RNAscope PLUS Assays

The RNAscope™ Plus smRNA-RNA Assay enables visualization of one small RNA (ASO, miRNA, siRNA) plus up to three mRNA markers.

Infographic RNAscope & gene therapy

miRNAscope Assay

The targeting siRNA was detected using the siRNA probe in the siRNA-treated mouse liver tissue samples and not in the vehicle tissue samples using the miRNAscope assay, indicating successful delivery of the siRNA-based therapy. Expression of target mRNA was detected using the RNAscope assay and appeared significantly reduced in the siRNA-treated tissue compared to vehicle-treated tissue suggesting successful knock-down of the target mRNA expression with the siRNA-based therapy.

RNAscope Assay data

RNAscope™ Plus small RNA-RNA Assay

Detection of Small RNAs and Target RNAs. The RNAscope Plus assay leverages the patented core technology that enables signal amplification and background suppression for multiplex fluorescent visualization of one small RNA with up to three mRNAs in formalin-fixed paraffin-embedded (FFPE) and fresh frozen samples.

On Demand Webinar

Multiomics spatial biodistribution visualization tool for RNAi and oligonucleotide-based therapies

 

Products & Services 

RNAscope Professional Assay Services

Our Professional Assay Services offer you direct access to the developers of RNAscope in situ hybridization (ISH) technology. Accelerate your journey in target validation, preclinical, and clinical development with the promise that we will provide consistent and trustworthy data to drive your projects forward. 

 

RNAscope ISH Technology - Product Formats

Multiomic Spatial Solutions for RNA and Protein Co-detection

 

RNAscope

 ~20 ZZ probe pairs  Designed to > 300 nt.

  • Detect transgene expression
  • Detect AAV vector DNA
  • Detect cellular marker mRNA or protein

Learn About RNAscope

BaseScope™

1-3 ZZ probe pairs

Designed to ~ 50-300 nt.

  • Detect codon-optimized genes,  splice variants, exon junctions
  • Detect up to single-nucleotide differences
  • Detect cellular marker mRNA or protein

Learn About BaseScope

miRNAscope™

Designed to ~17-50 nt.

  • Detect small non-coding RNAs (miRNA, ASO, siRNA)
  • Detect smRNA and protein

Learn About miRNAscope

RNAscope Plus

RNA species of varied sizes.

  • Visualize - 1 small RNA (ASO, miRNA, siRNA) + up to 3 mRNAs
  • Evaluate expression – of the target gene and cell specific marker genes
  • Detect 1 smRNA, 2 mRNA, and protein (IF)

Learn About RNAscope Plus

Resources for Oligonucleotide Therapy

Related Resources

Reference

  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.