ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 309 papers.
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Who cites it
309 citing papers in PubMed, 502 citations in OpenAlex.
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- Messenger RNA-encoded reporters for monitoring cellular stress and bioenergetics.Scientific reports · 2026Article
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- Enhancing mRNA stability and translational potential through tailored modifications at the 3' end.RSC chemical biology · 2026Article
- From Global Insights to Local Action: Bridging Vaccine Design and Manufacturing Gaps in H5N1 Pandemic Readiness.Vaccines · 2026Article
- Beyond the Sequence: Chemical and Topological Design and Innovations in mRNA Therapeutics.Chemical reviews · 2026Review
- From RNA to DNA: How Cargo Identity Reprograms Lipid Nanoparticle Architecture and Function.Advanced healthcare materials · 2026Review
- Rationale for multi-epitope TGFβ vaccination in pancreatic cancer: evidence from immunologic and clinical correlates.Signal transduction and targeted therapy · 2026Article
- Integrative bioinformatics approach to the mRNA vaccine development for the human papillomavirus using the capsid protein.Biology direct · 2026Article
- Navigating the next frontier in biomedicine: breakthroughs and insights in nucleic acid therapeutics.Chemical science · 2026Review
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- mRNA-based therapies: current progress and future prospects for treating kidney disease.Clinical and experimental nephrology · 2026Review
- mRNA vaccines in cancer immunotherapy: current progress and perspectives in solid tumors and hematologic malignancies.MedScience · 2026Review
- Smart dendrimer nanogels boost mRNA-based cancer therapy via synergistic glycolysis inhibition and immune activation.Bioactive materials · 2026Article
- Influenza A virus derived NS1 enhances translation of HPLC purified mRNA and interferon adjuvanted mRNA vaccination.Scientific reports · 2026Article
- Messenger RNA therapy in bone and joint diseases: Rationale, delivery systems, and applications.Biomaterials translational · 2026Article
249 more citing papers are in PubMed but not listed here.
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Authors and funding
7 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Being a transient carrier of genetic information, mRNA could be a versatile, flexible, and safe means for protein therapies. While recent findings highlight the enormous therapeutic potential of mRNA, evidence that mRNA-based protein therapies are feasible beyond small animals such as mice is still lacking. Previous studies imply that mRNA therapeutics require chemical nucleoside modifications to obtain sufficient protein expression and avoid activation of the innate immune system. Here we show that chemically unmodified mRNA can achieve those goals as well by applying sequence-engineered molecules. Using erythropoietin (EPO) driven production of red blood cells as the biological model, engineered Epo mRNA elicited meaningful physiological responses from mice to nonhuman primates. Even in pigs of about 20 kg in weight, a single adequate dose of engineered mRNA encapsulated in lipid nanoparticles (LNPs) induced high systemic Epo levels and strong physiological effects. Our results demonstrate that sequence-engineered mRNA has the potential to revolutionize human protein therapies.
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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 OpenQuestion graph.