ArticleMolecular therapy. Nucleic acids2025
Discovery and characterization of stereodefined PMO-gapmers targeting tau.
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 7 papers.
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Who cites it
7 citing papers in PubMed.
- Survivin-Targeting Antisense Oligonucleotides in Cancer Therapy.Molecules (Basel, Switzerland) · 2026Review
- The mechanisms and strategies for Alzheimer's disease prevention: An update.Acta pharmaceutica Sinica. B · 2026Review
- Advances in Analysis of Therapeutic Oligonucleotides with Chromatography Coupled to Mass Spectrometry.Analytical chemistry · 2026Review
- Stereocontrolled Synthesis of Dimethylamino Phosphorochloridate Monomers toward Stereopure Phosphorodiamidate Morpholino Oligonucleotides.The Journal of organic chemistry · 2026Article
- Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.Molecular therapy. Nucleic acids · 2026Article
- A single stereogenic center matters: Development of stereodefined anti-tau PMO-gapmers.Molecular therapy. Nucleic acids · 2025Article
- An Improved P(V) Thio-Oligonucleotide Synthesis Platform.Organic letters · 2025Article
Corrections and comments
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Authors and funding
26 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Antisense oligonucleotides (ASOs) are an important class of therapeutics to treat genetic diseases, and expansion of this modality to neurodegenerative disorders has been an active area of research. To realize chronic administration of ASO therapeutics to treat neurodegenerative diseases, new chemical modifications that improve activity and safety profiles are still needed. Furthermore, it is highly desirable to develop a single stereopure ASO with a defined activity and safety profile to avoid any efficacy and safety concerns due to the batch-to-batch variation in the composition of diastereomers. Here, a stereopure PMO-gapmer was developed as a new construct to improve safety and stability by installing charge-neutral PMOs at the wing region and by fully controlling phosphorus stereochemistries. The developed stereopure PMO-gapmer construct was applied to the discovery of ASO candidates for the reduction of microtubule-associated protein tau (
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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 Socratic graph.