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Nucleic acid delivery
Nucleic acid delivery
For nucleic-acid drugs, delivery often determines whether a sequence can produce sufficient expression or knockdown in the target tissue.
Screen siRNA, ASO, mRNA and AON candidates across SC, IV, IT, ICV, IM, IVT and intratympanic routes, with tissue-specific quantification.
Multi-candidate, multi-tissue qPCR comparison can identify the strongest candidate and delivery strategy before disease-model efficacy work.
- Liver · muscle · CNS · eye · earTarget tissues
- SC · IV · IT · ICV · IVTRoutes
- Expression / knockdownQuantified effect
Model strategy
Compare candidate sequences, routes, tissue distribution and expression or knockdown.
Match the model and study conditions to candidate mechanism, intervention stage and target tissue.
siRNA / ASO
Target-gene expression and knockdown
mRNA
Expression effect
Delivery route
SC / IV / IT / ICV / IVT matched to tissue
Evaluation endpoints
- 01Cross-candidate comparison
- 02Liver, muscle and peripheral tissue
- 03Brain regions, spinal cord and DRG
- 04Retina, cochlea and other tissue quantification
Representative data
SC subcutaneous injection · Liver rapid screening
Bar graph of relative liver mRNA expression (R.E.) in mice after subcutaneous (SC) injection of 3 mpk candidate siRNAs, comparing the PBS control group with 12 candidate groups in the same batch, with significance of differences between groups annotated.
IVT and intratympanic delivery | Retina and cochlea validation
Relative target gene mRNA expression in the retina, sclera-choroid complex and left/right cochlea after intravitreal (IVT) and intratympanic dosing; both local routes achieved significant knockdown.
Efficacy in Various Tissues following SC | Subcutaneous injection · Simultaneous distribution profile across 12 tissues
Bar graph of relative mRNA expression from simultaneous collection and quantification across 12 tissues after subcutaneous injection (heart, liver, spleen, lung, adrenal gland, gastrocnemius, quadriceps, diaphragm, iBAT, WAT, OVAT, MWAT), comparing the PBS and siRNA-1/siRNA-2 (5 mpk) groups.
Efficacy Results of IT and ICV | Intrathecal & intracerebroventricular injection · Spinal cord · Target brain regions · DRG target gene knockdown
Bar graph of relative mRNA expression in the spinal cord (lumbar/thoracic/cervical segments), multiple brain regions (hippocampus, thalamus, striatum, brainstem, cerebellum, cortex), heart/quadriceps/liver and DRG after intrathecal (IT) and intracerebroventricular (ICV) injection of 150 μg, comparing the PBS, IT and ICV groups.
Technical contact
Discuss your target, model or delivery question and learn about study plans, project scope and pricing.