ArticleMolecular therapy. Nucleic acids2019
Effective In Vivo Topical Delivery of siRNA and Gene Silencing in Intact Corneal Epithelium Using a Modified Cell-Penetrating Peptide.
Article in Molecular therapy. Nucleic acids, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 41 citations in OpenAlex.
- Transcriptome Analysis of TGFBI Knockdown vs Normal Corneal Epithelial Cells: Implications for TGFBI Corneal Dystrophy Treatment.Biochemical genetics · 2026Article
- Progress in Nanotechnology for Treating Ocular Surface Chemical Injuries: Reflecting on Advances in Ophthalmology.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- siRNA Treatment Enhances Collagen Fiber Formation in Tissue-Engineered Meniscus via Transient Inhibition of Aggrecan Production.Bioengineering (Basel, Switzerland) · 2024Article
- Suppressing Pro-Apoptotic Proteins by siRNA in Corneal Endothelial Cells Protects against Cell Death.Biomedicines · 2024Article
- Peptide cargo administration: current state and applications.Applied microbiology and biotechnology · 2023Review
- Potential therapeutic strategies for photoreceptor degeneration: the path to restore vision.Journal of translational medicine · 2022Review
- Deliver the promise: RNAs as a new class of molecular entities for therapy and vaccination.Pharmacology & therapeutics · 2022Review
- Gene Therapy in the Anterior Eye Segment.Current gene therapy · 2022Review
- PolyPurine Reverse Hoogsteen Hairpins Work as RNA Species for Gene Silencing.International journal of molecular sciences · 2021Article
- Peptidomimetics Therapeutics for Retinal Disease.Biomolecules · 2021Review
- A Novel Therapeutic Approach to Corneal Alkaline Burn Model by Targeting Fidgetin-Like 2, a Microtubule Regulator.Translational vision science & technology · 2021Article
- siRNA Therapeutics in Ocular Diseases.Methods in molecular biology (Clifton, N.J.) · 2021Review
- USP10 Targeted Self-Deliverable siRNA to Prevent Scarring in the Cornea.Molecular therapy. Nucleic acids · 2020Article
- Osteoblasts mineralization and collagen matrix are conserved upon specificMatrix biology plus · 2020Article
- Internalization mechanisms of cell-penetrating peptides.Beilstein journal of nanotechnology · 2020Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Autosomal dominantly inherited genetic disorders such as corneal dystrophies are amenable to allele-specific gene silencing with small interfering RNA (siRNA). siRNA delivered to the cornea by injection, although effective, is not suitable for a frequent long-term treatment regimen, whereas topical delivery of siRNA to the cornea is hampered by the eye surface's protective mechanisms. Herein we describe an attractive and innovative alternative for topical application using cell-penetrating peptide derivatives capable of complexing siRNA non-covalently and delivering them into the cornea. Through a rational design approach, we modified derivatives of a cell-penetrating peptide, peptide for ocular delivery (POD), already proved to diffuse into the corneal layers. These POD derivatives were able to form siRNA-peptide complexes (polyplexes) of size and ζ-potential similar to those reported able to undergo cellular internalization. Successful cytoplasmic release and gene silencing in vitro was obtained when an endosomal disruptor, chloroquine, was added. A palmitoylated-POD, displaying the best delivery properties, was covalently functionalized with trifluoromethylquinoline, an analog of chloroquine. This modified POD, named trifluoromethylquinoline-palmitoyl-POD (QN-Palm-POD), when complexed with siRNA and topically applied to the eye in vivo, resulted in up to 30% knockdown of luciferase reporter gene expression in the corneal epithelium. The methods developed within represent a valid standardized approach that is ideal for screening of a range of delivery formulations.
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