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Updated: Apr 19, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
Sequence-defined polymers for the delivery of oligonucleotides
1Pharmaceutical Biotechnology, Department of Pharmacy and Center for Nanoscience (CeNS), Ludwig-Maximilians-University, Munich, Germany.
Abstract:
Short synthetic oligonucleotides (ONs) are a group of therapeutic molecules with enormous clinical potential owing to their high specificity and ability to target the expression of virtually any single or group of genes. Clinical translation of ONs is hampered by the inadequate bioavailability in the target cells due to the substantial extracellular and intracellular barriers exposed to these molecules. Different cationic polymers have been successfully deployed for the delivery of ONs. However, heterogeneous nature of these classical polymers is not suitable for clinical applications and hence vectors with completely defined structure are required. In this review, we discuss recent advances with sequence-defined polymers and their application for the delivery of short ONs.
Insights
Sequence-defined polymers offer a promising solution for delivering therapeutic oligonucleotides (ONs), overcoming bioavailability challenges and advancing gene-targeting therapies for clinical use.
Area of Science:
- Biomaterials Science
- Gene Therapy
- Polymer Chemistry
Background:
- Short synthetic oligonucleotides (ONs) show high specificity for gene expression targeting, holding significant therapeutic potential.
- Clinical application of ONs is limited by poor bioavailability due to extracellular and intracellular barriers.
- Existing cationic polymers for ON delivery are heterogeneous and unsuitable for clinical use.
Purpose of the Study:
- To review recent advancements in sequence-defined polymers for oligonucleotide delivery.
- To highlight the potential of defined polymers in overcoming ON bioavailability issues.
Main Methods:
- Review of literature on sequence-defined polymers and their application in oligonucleotide delivery.
- Analysis of the structural advantages of sequence-defined polymers over classical polymers.
Main Results:
- Sequence-defined polymers offer a defined structure, addressing limitations of heterogeneous classical polymers.
- These advanced polymers show promise in enhancing the delivery and bioavailability of short ONs.
Conclusions:
- Sequence-defined polymers represent a significant advancement for the clinical translation of oligonucleotide therapeutics.
- The development of well-defined delivery vectors is crucial for unlocking the full potential of gene-targeting ONs.
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