Synthetic oligonucleotides such as small interfering RNAs (siRNAs) and anti-sense oligonucleotides (ASOs) can target specific genes and regulate their expression. These therapeutic agents can be designed to target aberrantly expressed genes in a wide range of disease conditions. The new miRNAscope™ assay can visualize and monitor the delivery and biodistribution of these synthetic RNA molecules to access the specificity and efficacy of these therapies.

Oncology

siRNAs and ASOs can be designed to selectively target oncogenes relevant to specific malignancies and downregulate their expression to control tumor growth.

Neuroscience

siRNAs and ASOs are highly promising tools for the clinical treatment of acute brain injury, neurodegenerative disorders and can potentially treat previously inaccessible drug targets implicated in neuromuscular and neurodegenerative disorders.

Immunology

siRNAs and ASOs can be used to target differentially expressed genes in patients with auto-immune disorders and mitigate disease progression.

Figure 1. 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.