ArticleMolecular therapy. Nucleic acids2025
Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotides
Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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Who cites it
4 citing papers in PubMed.
- Chemical Modification Strategies for Therapeutic Oligonucleotides: Mechanism Compatibility, Design Trade-Offs, and Translational Barriers.Molecules (Basel, Switzerland) · 2026Review
- The Versatile Applications of Antisense Oligonucleotides in Modern Medicine.International journal of molecular sciences · 2026Review
- Metabolic Dysfunction-Associated Steatotic Liver Disease and Emerging Oligonucleotide Therapies.International journal of biological sciences · 2026Review
- Design of bifunctional RNA-binding compounds that can modulate siRNA hydrophobicity for deep penetration and gene silencing in the retina.Molecular therapy. Nucleic acids · 2025Article
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Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Antisense oligonucleotides (ASOs), including splice-switching oligonucleotides (SSOs), are promising therapeutic approaches for targeting genetic defects. ASOs act in the nucleus and the cytosol to cleave mRNAs via the RNaseH1 mechanism (e.g., gapmers), while SSOs alter transcript splicing to restore or inhibit protein function. RNA interference (RNAi) is another approach to down-regulate gene expression via the RISC complex. However, a major challenge is the effective delivery of these nucleic acid-based therapeutics. Recent developments focus on enhancing cellular uptake and endosomal release, including the use of small-molecule endosomal escape enhancers (EEEs) such as chloroquine. Here, we disclose a next generation of EEEs, which efficiently enhance SSOs and gapmers
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