ArticleCommunications biology2022
Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides.
Article in Communications biology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 18 citations in OpenAlex.
- Engineered Brimonidine Tartrate Aminosomes as a Mucoadhesive Platform for Sustained Glaucoma Management: Optimization, In Vitro Characterization, and In Vivo Evaluation.Pharmaceutics · 2026Article
- Bioconjugates for improved delivery of oligonucleotide therapeutics to the central nervous system.Advanced drug delivery reviews · 2026Review
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- Review
- Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotidesMolecular therapy. Nucleic acids · 2025Article
- Formulation and Characterization of Novel Ionizable and Cationic Lipid Nanoparticles for the Delivery of Splice-Switching Oligonucleotides.Advanced materials (Deerfield Beach, Fla.) · 2025Article
- Unlocking Intracellular Protein Delivery by Harnessing Polymersomes Synthesized at Microliter Volumes using Photo-PISA.Advanced materials (Deerfield Beach, Fla.) · 2024Article
- Get Spliced: Uniting Alternative Splicing and Arthritis.International journal of molecular sciences · 2024Review
- Therapeutic Oligonucleotides: An Outlook on Chemical Strategies to Improve Endosomal Trafficking.Cells · 2023Review
- The cellular response to extracellular vesicles is dependent on their cell source and dose.Science advances · 2023Article
- Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides.Communications biology · 2022Article
Corrections and comments
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Authors and funding
21 authors at 6 institutions in 5 countries.
Funding
Abstract
The therapeutic and research potentials of oligonucleotides (ONs) have been hampered in part by their inability to effectively escape endosomal compartments to reach their cytosolic and nuclear targets. Splice-switching ONs (SSOs) can be used with endosomolytic small molecule compounds to increase functional delivery. So far, development of these compounds has been hindered by a lack of high-resolution methods that can correlate SSO trafficking with SSO activity. Here we present in-depth characterization of two novel endosomolytic compounds by using a combination of microscopic and functional assays with high spatiotemporal resolution. This system allows the visualization of SSO trafficking, evaluation of endosomal membrane rupture, and quantitates SSO functional activity on a protein level in the presence of endosomolytic compounds. We confirm that the leakage of SSO into the cytosol occurs in parallel with the physical engorgement of LAMP1-positive late endosomes and lysosomes. We conclude that the new compounds interfere with SSO trafficking to the LAMP1-positive endosomal compartments while inducing endosomal membrane rupture and concurrent ON escape into the cytosol. The efficacy of these compounds advocates their use as novel, potent, and quick-acting transfection reagents for antisense ONs.
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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.