ArticleBiomedicines2021
Novel Orthogonally Hydrocarbon-Modified Cell-Penetrating Peptide Nanoparticles Mediate Efficient Delivery of Splice-Switching Antisense Oligonucleotides In Vitro and In Vivo.
Article in Biomedicines, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed.
- 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
- 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
- Amphipathic Octenyl-Alanine Modified Peptides Mediate Effective siRNA Delivery.Journal of peptide science : an official publication of the European Peptide Society · 2025Article
- Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotidesMolecular therapy. Nucleic acids · 2025Article
- Innovative dual-functional hybrid cationic PEGylated proniosomes as a smart nano-platform for Boosted vaginal delivery: multi-levelFrontiers in pharmacology · 2025Article
- mCherry on Top: A Positive Read-Out Cellular Platform for Screening DMD Exon Skipping Xenopeptide-PMO Conjugates.Bioconjugate chemistry · 2023Article
- Choosing an Optimal Solvent Is Crucial for Obtaining Cell-Penetrating Peptide Nanoparticles with Desired Properties and High Activity in Nucleic Acid Delivery.Pharmaceutics · 2023Article
- Oligonucleotides-Based Therapeutics.Biomedicines · 2021Article
Corrections and comments
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Authors and funding
18 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Splice-switching therapy with splice-switching oligonucleotides (SSOs) has recently proven to be a clinically applicable strategy for the treatment of several mis-splice disorders. Despite this, wider application of SSOs is severely limited by the inherently poor bioavailability of SSO-based therapeutic compounds. Cell-penetrating peptides (CPPs) are a class of drug delivery systems (DDSs) that have recently gained considerable attention for improving the uptake of various oligonucleotide (ON)-based compounds, including SSOs. One strategy that has been successfully applied to develop effective CPP vectors is the introduction of various lipid modifications into the peptide. Here, we repurpose hydrocarbon-modified amino acids used in peptide stapling for the orthogonal introduction of hydrophobic modifications into the CPP structure during peptide synthesis. Our data show that α,α-disubstituted alkenyl-alanines can be successfully utilized to introduce hydrophobic modifications into CPPs to improve their ability to formulate SSOs into nanoparticles (NPs), and to mediate high delivery efficacy and tolerability both in vitro and in vivo. Conclusively, our results offer a new flexible approach for the sequence-specific introduction of hydrophobicity into the structure of CPPs and for improving their delivery properties.
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