ArticlePharmaceutics2019
Sugar and Polymer Excipients Enhance Uptake and Splice-Switching Activity of Peptide-Dendrimer/Lipid/Oligonucleotide Formulations.
Article in Pharmaceutics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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.
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Who cites it
16 citing papers in PubMed, 16 citations in OpenAlex.
- Optimizing nucleic acid delivery using PF14 peptide and lipid nanoparticle systems.International journal of pharmaceutics: X · 2026Article
- Engineered Brimonidine Tartrate Aminosomes as a Mucoadhesive Platform for Sustained Glaucoma Management: Optimization, In Vitro Characterization, and In Vivo Evaluation.Pharmaceutics · 2026Article
- Freeze-Drying in Sucrose Followed by Cryomilling Enables the Formulation of sa-mRNA-LNP Powders for Inhalation.Pharmaceutics · 2026Article
- Advanced hybrid-stabilized cinacalcet nanocrystals for enhanced oral bioavailability: formulation and integratedFrontiers in pharmacology · 2026Article
- Ketoprofen-loaded quatsomes as a smart repurposed antifungal therapy for vaginal infections: formulation, characterization, and microbiological evaluation.Frontiers in pharmacology · 2026Article
- Innovative hyaluronic acid-decorated quatsomes for enhanced renal targeting: design, optimization andJournal of pharmacy & pharmaceutical sciences : a publication of the Canadian Society for Pharmaceutical Sciences, Societe canadienne des sciences pharmaceutiques · 2026Article
- Novel hybrid tri-polymer hyalurosomes: unlocking next-generation trans-tympanic therapeutics through multi-Scale evaluations.International journal of pharmaceutics: X · 2025Article
- Revolutionary hyaluronic acid-modified edge-activated spanlastics as a novel approach to boost Hepatoprotective activity of Curcumin: Optimization, biochemical analysis andInternational journal of pharmaceutics: X · 2025Article
- Accelerated Endosomal Escape of Splice-Switching Oligonucleotides Enables Efficient Hepatic Splice Correction.ACS applied materials & interfaces · 2025Article
- Application of Lyophilized Gene-Delivery Formulations to Dental Implant Surfaces: Non-Cariogenic Lyoprotectant Preserves Transfection Activity of Polyplexes Long-Term.Journal of pharmaceutical sciences · 2023Article
- Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides.Communications biology · 2022Article
- Internalisation and Biological Activity of Nucleic Acids Delivering Cell-Penetrating Peptide Nanoparticles Is Controlled by the Biomolecular Corona.Pharmaceuticals (Basel, Switzerland) · 2021Article
- Efficient Peptide-Mediated In Vitro Delivery of Cas9 RNP.Pharmaceutics · 2021Article
- Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects.The AAPS journal · 2021Review
- Non-Viral Gene Delivery Systems.Pharmaceutics · 2021Article
- Lipophilic Peptide Dendrimers for Delivery of Splice-Switching Oligonucleotides.Pharmaceutics · 2021Article
Corrections and comments
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Authors and funding
10 authors at 2 institutions in 4 countries.
Funding
Abstract
Non-viral transfection vectors are commonly used for oligonucleotide (ON) delivery but face many challenges before reaching the desired compartments inside cells. With the support of additional compounds, it might be more feasible for a vector to endure the barriers and achieve efficient delivery. In this report, we screened 18 different excipients and evaluated their effect on the performance of peptide dendrimer/lipid vector to deliver single-stranded, splice-switching ONs under serum conditions. Transfection efficiency was monitored in four different reporter cell lines by measuring splice-switching activity on RNA and protein levels. All reporter cell lines used had a mutated human β-globin intron 2 sequence interrupting the luciferase gene, which led to an aberrant splicing of luciferase pre-mRNA and subsidence of luciferase protein translation. In the HeLa Luc/705 reporter cell line (a cervical cancer cell line), the lead excipients (Polyvinyl derivatives) potentiated the splice-switching activity up to 95-fold, compared to untreated cells with no detected cytotoxicity. Physical characterization revealed that lead excipients decreased the particle size and the zeta potential of the formulations. In vivo biodistribution studies emphasized the influence of formulations as well as the type of excipients on biodistribution profiles of the ON. Subsequently, we suggest that the highlighted impact of tested excipients would potentially assist in formulation development to deliver ON therapeutics in pre-clinical and clinical settings.
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What OpenQuestion holds
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.