ArticleJournal of controlled release : official journal of the Controlled Release Society2014
In vivo gene silencing following non-invasive siRNA delivery into the skin using a novel topical formulation.
Article in Journal of controlled release : official journal of the Controlled Release Society, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 40 citations in OpenAlex.
- Nanoparticle-delivered miR-486-5p inhibits HNanoscale advances · 2026Article
- Nomilin Rewires mTOR-Driven Immunometabolism via DDIT4 to Restrain Psoriasis.International journal of biological sciences · 2026Article
- Activation of G protein gated inwardly rectifying potassium (GIRK) channels in keratinocytes mediates peripheral kappa opioid receptor-mediated antinociception.Neuropharmacology · 2025Article
- Approaches and applications in transdermal and transpulmonary gene drug delivery.Frontiers in bioengineering and biotechnology · 2024Review
- Passive siRNA transfection method for gene knockdown in air-liquid interface airway epithelial cell cultures.American journal of physiology. Lung cellular and molecular physiology · 2021Article
- Development of a Corneal Bioluminescence Mouse for Real-Time In Vivo Evaluation of Gene Therapies.Translational vision science & technology · 2020Article
- USP10 Targeted Self-Deliverable siRNA to Prevent Scarring in the Cornea.Molecular therapy. Nucleic acids · 2020Article
- Allele-Specific Small Interfering RNA Corrects Aberrant Cellular Phenotype in Keratitis-Ichthyosis-Deafness Syndrome Keratinocytes.The Journal of investigative dermatology · 2020Article
- Fractional Laser-Mediated siRNA Delivery for Mitigating Psoriasis-like Lesions via IL-6 Silencing.Molecular therapy. Nucleic acids · 2020Article
- Rekindling RNAi Therapy: Materials Design Requirements for In Vivo siRNA Delivery.Advanced materials (Deerfield Beach, Fla.) · 2019Review
- Effective In Vivo Topical Delivery of siRNA and Gene Silencing in Intact Corneal Epithelium Using a Modified Cell-Penetrating Peptide.Molecular therapy. Nucleic acids · 2019Article
- Is the Fractional Laser Still Effective in Assisting Cutaneous Macromolecule Delivery in Barrier-Deficient Skin? Psoriasis and Atopic Dermatitis as the Disease Models.Pharmaceutical research · 2018Article
- Construction of histidine-containing hydrocarbon stapled cell penetrating peptides forChemical science · 2018Article
- Nucleophosmin modulates the alleviation of atopic dermatitis caused by the marine-derived compound dihydroaustrasulfone alcohol.Experimental & molecular medicine · 2018Article
- siRNA-Mediated RNA Interference in Precision-Cut Tissue Slices Prepared from Mouse Lung and Kidney.The AAPS journal · 2017Article
- Advances in the Application and Impact of MicroRNAs as Therapies for Skin Disease.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2017Review
- Non-Invasive Intravital Imaging of siRNA-Mediated Mutant Keratin Gene Repression in Skin.Molecular imaging and biology · 2016Article
- Cardiac Gene Expression Knockdown Using Small Inhibitory RNA-Loaded Microbubbles and Ultrasound.PloS one · 2016Article
- Dermal delivery of HSP47 siRNA with NOX4-modulating mesoporous silica-based nanoparticles for treating fibrosis.Biomaterials · 2015Article
- Distinct Impact of Two Keratin Mutations Causing Epidermolysis Bullosa Simplex on Keratinocyte Adhesion and Stiffness.The Journal of investigative dermatology · 2015Article
Corrections and comments
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Therapeutics based on short interfering RNAs (siRNAs), which act by inhibiting the expression of target transcripts, represent a novel class of potent and highly specific next-generation treatments for human skin diseases. Unfortunately, the intrinsic barrier properties of the skin combined with the large size and negative charge of siRNAs make epidermal delivery of these macromolecules quite challenging. To help evaluate the in vivo activity of these therapeutics and refine delivery strategies we generated an innovative reporter mouse model that predominantly expresses firefly luciferase (luc2p) in the paw epidermis--the region of murine epidermis that most closely models the tissue architecture of human skin. Combining this animal model with state-of-the-art live animal imaging techniques, we have developed a real-time in vivo analysis work-flow that has allowed us to compare and contrast the efficacies of a wide range nucleic acid-based gene silencing reagents in the skin of live animals. While inhibition was achieved with all of the reagents tested, only the commercially available "self-delivery" modified Accell-siRNAs (Dharmacon) produced potent and sustained in vivo gene silencing. Together, these findings highlight just how informative reliable reporter mouse models can be when assessing novel therapeutics in vivo. Using this work-flow, we developed a novel clinically-relevant topical formulation that facilitates non-invasive epidermal delivery of unmodified and "self-delivery" siRNAs. Remarkably, a sustained >40% luc2p inhibition was observed after two 1-hour treatments with Accell-siRNAs in our topical formulation. Importantly, our ability to successfully deliver siRNA molecules topically brings these novel RNAi-based therapeutics one-step closer to clinical use.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.