ReviewNucleic acid therapeutics2018
GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics.
Review in Nucleic acid therapeutics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 355 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
355 citing papers in PubMed, 2 syntheses or guidelines pooled it, 632 citations in OpenAlex.
- Safety profile of small interfering RNA therapeutics: a systematic review and meta-analysis of randomized controlled trials.Frontiers in drug safety and regulation · 2026Pooled it
- Model-based meta-analysis to quantify the effects of short interfering RNA therapeutics on hepatitis B surface antigen turnover in hepatitis B-infected mice.CPT: pharmacometrics & systems pharmacology · 2024Pooled it
- siRNA did not fail in cancer. It was given the wrong job.Molecular therapy. Nucleic acids · 2026Article
- An EGFR-Targeted Fusogenic Tandem Peptide for siRNA Delivery in Glioblastoma.Pharmaceutics · 2026Article
- RNA-Based Therapeutics in Genetic Neurodevelopmental Disorders: Bridging Molecular Genetics and Precision Medicine.International journal of molecular sciences · 2026Review
- Precision Lipid Management in the Era of Biotechnology and Artificial Intelligence: From Gene Editing to Smart Drug Delivery.Therapeutic innovation & regulatory science · 2026Review
- Engineering the Future of Precision Medicine: A Comprehensive Guide to RNA Therapeutics.Current issues in molecular biology · 2026Review
- Zilebesiran, a Small Interfering RNA Therapeutic Targeting Angiotensinogen: Mechanism, Clinical Evidence, Safety, and Implementation Considerations.Life (Basel, Switzerland) · 2026Review
- Targeting Spatiotemporal Heterogeneity of oncomiRNAs: A New Frontier in Cancer Therapy.Cancer reports (Hoboken, N.J.) · 2026Review
- Potent and Sustained Lowering of Serum Uric Acid by YJH-012-D, a GalNAc-Conjugated siRNA Targeting Xanthine Dehydrogenase.Pharmacology research & perspectives · 2026Article
- Pharmacokinetic/Pharmacodynamic Translation and Model-Informed Drug Development for Oligonucleotide Therapeutics.Journal of clinical pharmacology · 2026Review
- AI-Guided Long Non-Coding RNA Target Discovery for Precision Medicine: Integrating GWAS, Multi-Omics, Experimental Validation, and RNA Therapeutics.Biomedicines · 2026Review
- 1'-Sugar-Tethered Cholesterol-Modified siRNAs with Variable Linker Lengths for Carrier-Free Delivery and Enhanced RNAi Efficiency.ACS omega · 2026Article
- Genetic or pharmacological inhibition of hepatic TMEM141 attenuates MASH and fibrosis via the ROS-HNF4α signaling pathway.Nature communications · 2026Article
- Review
- Biologics for cardiovascular diseases: from bench to bedside.Signal transduction and targeted therapy · 2026Review
- The Versatile Applications of Antisense Oligonucleotides in Modern Medicine.International journal of molecular sciences · 2026Review
- Programming Brain Cell-Type-Selective Delivery In Vivo with Transporter-Guided Therapeutics.bioRxiv : the preprint server for biology · 2026Article
- Investigating theBioconjugate chemistry · 2026Article
- Therapeutic Strategies for Hyperuricemia: From Small-Molecule Inhibitors to RNA Therapeutics.ACS pharmacology & translational science · 2026Review
295 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 at 1 institution in 1 country.
Funding
Abstract
Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease. However, before siRNAs can become drugs, they must overcome a billion years of evolutionary defenses designed to keep invading RNAs on the outside cells from getting to the inside of cells. Not surprisingly, significant effort has been placed in developing a wide array of delivery technologies. Foremost of these has been the development of N-acetylgalactosamine (GalNAc) siRNA conjugates for delivery to liver. Tris-GalNAc binds to the Asialoglycoprotein receptor that is highly expressed on hepatocytes resulting in rapid endocytosis. While the exact mechanism of escape across the endosomal lipid bilayer membrane remains unknown, sufficient amounts of siRNAs enter the cytoplasm to induce robust, target selective RNAi responses in vivo. Multiple GalNAc-siRNA conjugate clinical trials, including two phase III trials, are currently underway by three biotech companies to treat a wide variety of diseases. GalNAc-siRNA conjugates are a simple solution to the siRNA delivery problem for liver hepatocytes and have shown the RNAi (and antisense oligonucleotide) field the path forward for targeting other tissue types.
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.