ReviewNucleic acids research2023
Chemistry, structure and function of approved oligonucleotide therapeutics.
Review in Nucleic acids research, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 425 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
425 citing papers in PubMed.
- Barcoded oligonucleotide system (BOLT) for targeted organ delivery.Bioactive materials · 2027Article
- Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment.RNA biology · 2026Review
- Article
- Broad-spectrum aptamers: recent advances in selection strategies, structural mechanisms, and biosensing applications.Mikrochimica acta · 2026Review
- Chemically stabilized long antisense oligonucleotides perturb RNA exosome function.Nucleic acids research · 2026Article
- Targeting SUV39H2 exon skipping reverses resistance to CDK4/6 inhibitors via transcriptional activation of p21.Science advances · 2026Article
- Exploring the Potential of PhosphoramideACS omega · 2026Article
- Article
- Nucleoside drives the self-assembly and enables delivery of phosphate containing therapeutics.Materials today. Bio · 2026Article
- A versatile strategy for oligonucleotide functionalization via on-support phosphitylation.Nature chemistry · 2026Article
- Triazole-Linked Pyrimidine Nucleoside Dimers: Design, Synthesis, and Applications in Medicinal and Nucleic Acid Chemistry. Part IV.Chemical biology & drug design · 2026Article
- Synthesis of Oligonucleotide Conjugates Employing a Diselenide Linker.Chemistry (Weinheim an der Bergstrasse, Germany) · 2026Article
- On-support phosphitylation for oligonucleotide modification.Nature chemistry · 2026Article
- A 4'-Thioribouridine Modification Mimics 2'-O-Methoxyethyl in ASO-Like 20-Mers Targeting PTEN Knockdown.Chembiochem : a European journal of chemical biology · 2026Article
- Silencing with precision: In silico strategies for antisense oligonucleotide engineering.Molecular biology reports · 2026Review
- RNA therapeutics: current status and future directions.Signal transduction and targeted therapy · 2026Review
- Photocatalytic synthesis of phosphorothioates by sulfur transfer from thiosulfate.Chemical science · 2026Article
- Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system.Nucleic acids research · 2026Article
- An Enzymatic Platform for Late-Stage (Radio)isotope Labelling of Oligonucleotides With Methyltransferases.Angewandte Chemie (International ed. in English) · 2026Article
- The evolving landscape of drug targets.Nature reviews. Drug discovery · 2026Review
365 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi) and an RNA aptamer against a protein. Among the diseases targeted by this new class of drugs are homozygous familial hypercholesterolemia, spinal muscular atrophy, Duchenne muscular dystrophy, hereditary transthyretin-mediated amyloidosis, familial chylomicronemia syndrome, acute hepatic porphyria, and primary hyperoxaluria. Chemical modification of DNA and RNA was central to making drugs out of oligonucleotides. Oligonucleotide therapeutics brought to market thus far contain just a handful of first- and second-generation modifications, among them 2'-fluoro-RNA, 2'-O-methyl RNA and the phosphorothioates that were introduced over 50 years ago. Two other privileged chemistries are 2'-O-(2-methoxyethyl)-RNA (MOE) and the phosphorodiamidate morpholinos (PMO). Given their importance in imparting oligonucleotides with high target affinity, metabolic stability and favorable pharmacokinetic and -dynamic properties, this article provides a review of these chemistries and their use in nucleic acid therapeutics. Breakthroughs in lipid formulation and GalNAc conjugation of modified oligonucleotides have paved the way to efficient delivery and robust, long-lasting silencing of genes. This review provides an account of the state-of-the-art of targeted oligo delivery to hepatocytes.
Indexed as
Identifiers
What OpenQuestion holds
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.