ReviewClinical and translational science2019
Targeting RNA: A Transformative Therapeutic Strategy.
Review in Clinical and translational science, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 69 papers, 2 of them syntheses that pooled it.
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
69 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.Pharmacological reviews · 2020Pooled it
- Pharmacological PTEN inhibition: potential clinical applications and effects in tissue regeneration.Regenerative medicine · 2020Pooled it
- Treatment with Volanesorsen, a 2'-O-Methoxyethyl-Modified Antisense Oligonucleotide TargetingNucleic acid therapeutics · 2020Trial
- Photocatalytic synthesis of phosphorothioates by sulfur transfer from thiosulfate.Chemical science · 2026Article
- Article
- Therapeutic modulation of HIV-1 using miRNAs and lncRNAs: from bench to bedside.Virology journal · 2026Review
- Advancing regenerative medicine with RNA nanotechnology for chronic and end organ diseases.Communications medicine · 2026Review
- Antibody-Drug Conjugates (ADCs): A Review of Structural Design, Technological Evolution, and Future Perspectives.Molecules (Basel, Switzerland) · 2026Review
- mRNA vaccines and SiRNAs targeting cancer immunotherapy: challenges and opportunities.Discover oncology · 2025Review
- Article
- Harnessing RNA therapeutics: novel approaches and emerging strategies for cardiovascular disease management.Frontiers in cardiovascular medicine · 2025Review
- Exploring precision therapeutics: computational design of antisense oligonucleotides targeting AXL gene transcripts in multiple sclerosis treatment management.Frontiers in chemistry · 2025Article
- Mechanistic insights into ASO-RNA complexation: Advancing antisense oligonucleotide design strategies.Molecular therapy. Nucleic acids · 2024Article
- Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery.Molecular therapy. Nucleic acids · 2024Review
- RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges.Molecular therapy. Nucleic acids · 2024Review
- Targeting Lipoprotein(a): Can RNA Therapeutics Provide the Next Step in the Prevention of Cardiovascular Disease?Cardiology and therapy · 2024Review
- Review
- Targeting RNA with synthetic oligonucleotides: Clinical success invites new challenges.Cell chemical biology · 2024Review
- Antisense oligonucleotides and their applications in rare neurological diseases.Frontiers in neuroscience · 2024Review
- A Multistakeholder Perspective on Advancing Individualized Therapeutics.Clinical pharmacology and therapeutics · 2023Review
9 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
The therapeutic pathways that modulate transcription mechanisms currently include gene knockdown and splicing modulation. However, additional mechanisms may come into play as more understanding of molecular biology and disease etiology emerge. Building on advances in chemistry and delivery technology, oligonucleotide therapeutics is emerging as an established, validated class of drugs that can modulate a multitude of genetic targets. These targets include over 10,000 proteins in the human genome that have hitherto been considered undruggable by small molecules and protein therapeutics. The approval of five oligonucleotides within the last 2 years elicited unprecedented excitement in the field. However, there are remaining challenges to overcome and significant room for future innovation to fully realize the potential of oligonucleotide therapeutics. In this review, we focus on the translational strategies encompassing preclinical evaluation and clinical development in the context of approved oligonucleotide therapeutics. Translational approaches with respect to pharmacology, pharmacokinetics, cardiac safety evaluation, and dose selection that are specific to this class of drugs are reviewed with examples. The mechanism of action, chemical evolution, and intracellular delivery of oligonucleotide therapies are only briefly reviewed to provide a general background for this class of drugs.
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.