ReviewMolecular therapy : the journal of the American Society of Gene Therapy2019
Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 65 papers, 1 of them a synthesis 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
65 citing papers in PubMed, 1 synthesis or guideline pooled it.
- SALL4 and microRNA: The Role of Let-7.Genes · 2021Pooled it
- Codon-optimizedMolecular therapy. Nucleic acids · 2026Article
- mRNA therapy improves the composition and motility in CCDC40-deficient cilia in vitro and in vivo.American journal of respiratory cell and molecular biology · 2026Article
- mRNA Lipid Nanoparticles for Cell Engineering in Vivo and in Vitro: Current Applications and Future Directions.MedComm · 2026Review
- Article
- Structure-Based Modeling of Environment-Dependent Protonation States Across LNP Formulations with Atomistic CpHMD.Molecular pharmaceutics · 2025Article
- Physiologically Based Pharmacokinetic Modeling and Simulation in Regulatory Review: US FDA CBER Experience and Perspectives.The AAPS journal · 2025Review
- Review
- Clinical development of therapeutic mRNA applications.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- mRNA-1273 is placenta-permeable and immunogenic in the fetus.Molecular therapy. Nucleic acids · 2025Article
- UTR-Insight: integrating deep learning for efficient 5' UTR discovery and design.BMC genomics · 2025Article
- The therapeutic landscape of citrin deficiency.Journal of inherited metabolic disease · 2024Review
- Protein is expressed in all major organs after intravenous infusion of mRNA-lipid nanoparticles in swine.Molecular therapy. Methods & clinical development · 2024Article
- mRNA vaccine development and applications: A special focus on tumors (Review).International journal of oncology · 2024Review
- Accelerating ionizable lipid discovery for mRNA delivery using machine learning and combinatorial chemistry.Nature materials · 2024Article
- Optimizing 5'UTRs for mRNA-delivered gene editing using deep learning.Nature communications · 2024Article
- Article
- Assembling the RNA therapeutics toolbox.Medical review (2021) · 2024Review
- Liquid crystalline inverted lipid phases encapsulating siRNA enhance lipid nanoparticle mediated transfection.Nature communications · 2024Article
- Inhalable dry powder product (DPP) of mRNA lipid nanoparticles (LNPs) for pulmonary delivery.Drug delivery and translational research · 2024Article
5 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
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Promising improvements in the field of transcript therapeutics have clearly enhanced the potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are engineered to replace mutated mRNAs and to be immunologically inconspicuous and highly stable while maximizing protein expression. Approaches to deliver mRNA into the cellular cytoplasm safely and efficiently have been further developed so that two mRNA-based approaches replacing vascular endothelial growth factor (VEGF) and cystic fibrosis transmembrane conductance regulator (CFTR) have now made it into clinical trials. These studies bring mRNA therapeutics for protein replacement therapy closer to clinical realization. Herein, we provide an overview of preclinical and clinical developments of mRNA therapeutics for liver diseases.
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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.