The DOI matches its Crossref record; 27 of the 27 references it cites were found in a registry or an index.
Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
Abstract
Current viral vectors for gene therapy are associated with serious safety concerns, including leukemogenesis, and nonviral vectors are limited by low gene transfer efficiency. Here we investigate the therapeutic utility of chemically modified mRNA as an alternative to DNA-based gene therapy. A combination of nucleotide modifications abrogates mRNA interaction with Toll-like receptor (TLR)3, TLR7, TLR8 and retinoid-inducible gene I (RIG-I), resulting in low immunogenicity and higher stability in mice. A single intramuscular injection of modified murine erythropoietin mRNA raises the average hematocrit in mice from 51.5% to 64.2% after 28 days. In a mouse model of a lethal congenital lung disease caused by a lack of surfactant protein B (SP-B), twice weekly local application of an aerosol of modified SP-B mRNA to the lung restored 71% of the wild-type SP-B expression, and treated mice survived until the predetermined end of the study after 28 days.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“Substitution of chemically modified nucleotides in messenger ribonucleic acid (mRNA) enhances stability and transfection capability, and decreases immunogenicity1–3”
From Complete substitution with modified nucleotides in self-amplifying RNA suppresses the interferon response and increases potency (2025) · cites this paper as [2]
In the citing paper
“Conclusive data have also been presented for mRNA as a platform for protein (replacement) therapies (Kormann et al, 2011; Kariko et al, 2012; Zangi et al, 2013; Thess et al, 2015; Balmayor et al, 2016).”
From mRNA mediates passive vaccination against infectious agents, toxins, and tumors (2017) · cites this paper as Kormann et al, 2011
In the citing paper
“Whereas unmodified Epo mRNA was immunostimulatory and failed to induce sufficient protein expression in mice, a few micrograms of nucleoside-modified mRNA were enough to achieve detectable protein expression and physiological effects upon direct administration to murine muscle.”
From Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals (2015) · cites this paper as [26]
In the citing paper
“Our work principally confirms that conventional unmodified mRNAs are inferior to molecules harboring specific nucleoside modifications as suggested by various recent reports,23,24,25,26,27 however, the picture changes dramatically with a sophisticated sequence-engineering.”
From Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals (2015) · cites this paper as [26]
In the citing paper
“Two independent studies on erythropoietin-encoding mRNA found a superiority of nucleoside-modified compared to unmodified mRNA.”
From Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals (2015) · cites this paper as [26]
In the citing paper
“While local administrations may be possible without the need for an mRNA delivery system,26,42”
From Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals (2015) · cites this paper as [26]
In the citing paper
“Kormann et al., demonstrated that 2 weeks following injection of nucleoside-modified mRNA containing 25% thio-uridine and 25% 5-methylcytidine resulted in Epo levels five times greater than that in untreated animals.”
From Modifications of mRNA vaccine structural elements for improving mRNA stability and translation efficiency (2022) · cites this paper as Kormann et al. 2011
In the citing paper
“Unmodified mRNA, on the other hand, showed no significant alterations and merely elicited a considerable immunological response (Kormann et al. 2011).”
From Modifications of mRNA vaccine structural elements for improving mRNA stability and translation efficiency (2022) · cites this paper as Kormann et al. 2011
In the citing paper
“In another study by Kormann and colleagues, translation was maximized and innate immune response was reduced in a mouse lung disease model by introducing chemically-modified nucleotides during in vitro transcription of mRNA (IVT-mRNA), coding for surfactant protein B (SP-B) with a 25% replacement of cytidine and uridine by modified 5-methyl cytidine (5-methyl CTP) and 2-thio uridine (2-thio UTP),”
From Human cellular CYBA UTR sequences increase mRNA translation without affecting the half-life of recombinant RNA transcripts (2016) · cites this paper as [19]
In the citing paper
“Therefore, chemists have creatively modified nucleic acid bases, sugar rings, 3 ‘and 5’ ends. These modifications are resistant to nuclease degradation, reduce immunogenicity and improve the interaction with target cells.311”
From Smart nanoparticles for cancer therapy (2023) · cites this paper as [311]
In the citing paper
“Studies have shown that when poly(A) tail size increases to 120 bp, the expression level of the corresponding protein increases accordingly. However, when poly(A) tail size is greater than 120 bp, the expression level of the corresponding protein did not increase.152”
From Advances in COVID-19 mRNA vaccine development (2022) · cites this paper as [152]
In the citing paper
“Kormann and colleagues152 replaced 25% of mRNA cytosine with m5C and 25% of uridine with s2U, which improved mRNA stability and increased protein translation in mice.”
From Advances in COVID-19 mRNA vaccine development (2022) · cites this paper as [152]
In the citing paper
“In contrast, no significant changes were detected with unmodified mRNA, which only evoked a substantial immune activation [44].”
From Three decades of messenger RNA vaccine development (2019) · cites this paper as [44]
In the citing paper
“In a therapeutic setting, Kormann et al., demonstrated that nucleoside-modified mRNA encoding erythropoietin (Epo), in which 25% thio-uridine and 25% 5-methylcytidine was incorporated, resulted in five times higher Epo levels compared to untreated mice, two weeks after i.m. administration.”
From Three decades of messenger RNA vaccine development (2019) · cites this paper as [44]
Citations
Cites
Pfaffl, M. W. (2002) · Nucleic Acids Research · doi:10.1093/nar/30.9.e36 · Cited by 7,377
Karikó, K., Buckstein, M., Ni, H., & Weissman, D. (2005) · Immunity · doi:10.1016/j.immuni.2005.06.008 · Cited by 2,728
Warren, L., Manos, P. D., Ahfeldt, T., Loh, Y.-H., Li, H., Lau, F., Ebina, W., Mandal, P. K., Smith, Z. D., Meissner, A., et al. (2010) · Cell Stem Cell · doi:10.1016/j.stem.2010.08.012 · Cited by 2,662
Effect of gene therapy on visual function in Leber’s congenital amaurosis
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924
Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
Hacein‐Bey‐Abina, S., Hauer, J., Lim, A., Pïcard, C., Wang, G. P., Berry, C. C., Martinache, C., Rieux‐Laucat, F., Latour, S., Belohradsky, B. H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000164 · Cited by 654
Deficiency of Pulmonary Surfactant Protein B in Congenital Alveolar Proteinosis
Nogee, L. M., deMello, D. E., Dehner, L. P., & Colten, H. R. (1993) · New England Journal of Medicine · doi:10.1056/nejm199302113280606 · Cited by 627
Holtkamp, S., Kreiter, S., Selmi, A., Simon, P., Koslowski, M., Huber, C., Türeci, O., & Sahin, U. (2006) · Blood · doi:10.1182/blood-2006-04-015024 · Cited by 625
Hydrophobic Surfactant Proteins in Lung Function and Disease
Whitsett, J. A., & Weaver, T. E. (2002) · New England Journal of Medicine · doi:10.1056/nejmra022387 · Cited by 519
A randomized controlled study of iron supplementation in patients treated with erythropoietin
Macdougall, I. C., Tucker, B., Thompson, J., Tomson, C., Baker, L. R. I., & Raine, A. E. G. (1996) · Kidney International · doi:10.1038/ki.1996.487 · Cited by 415
Targeted delivery of magnetic aerosol droplets to the lung
Dames, P., Gleich, B., Flemmer, A. W., Hajek, K., Seidl, N., Wiekhorst, F., Eberbeck, D., Bittmann, I., Bergemann, C., Weyh, T., et al. (2007) · Nature Nanotechnology · doi:10.1038/nnano.2007.217 · Cited by 382
Self-citation
Cited by
Sorted by citations
Items 401–500 of 731
Ohto, T., Konishi, M., Tanaka, H., Onomoto, K., Yoneyama, M., Nakai, Y., Tange, K., Yoshioka, H., & Akita, H. (2019) · Biological and Pharmaceutical Bulletin · doi:10.1248/bpb.b18-00783 · Cited by 29
Vasculature organotropism in drug delivery
Amruta, A., Iannotta, D., Cheetham, S. W., Lammers, T., & Wolfram, J. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.115054 · Cited by 29
Mandke, R., & Singh, J. (2011) · Pharmaceutical Research · doi:10.1007/s11095-011-0616-1 · Cited by 29
NIPBL Controls RNA Biogenesis to Prevent Activation of the Stress Kinase PKR
Yuen, K. C. Y., Xu, B., Krantz, I. D., & Gerton, J. L. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.12.012 · Cited by 29
Leyman, B., Huysmans, H., Cafferty, S. M., Combes, F., Cox, E., Devriendt, B., & Sanders, N. N. (2018) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.7b00722 · Cited by 30
Kim, J., Mukherjee, A., Nelson, D., Jozic, A., & Sahay, G. (2020) · doi:10.1101/2020.07.24.205583 · Cited by 28
Synthetic macromolecular switches for precision control of therapeutic cell functions
Teixeira, A. P., & Fussenegger, M. (2024) · Nature Reviews Bioengineering · doi:10.1038/s44222-024-00235-9 · Cited by 28
Progress and Challenges in HIV-1 Vaccine Research: A Comprehensive Overview
Boomgarden, A. C., & Upadhyay, C. (2025) · Vaccines · doi:10.3390/vaccines13020148 · Cited by 28
PTEN-mRNA engineered mesenchymal stem cell-mediated cytotoxic effects on U251 glioma cells
Guo, X., Hu, Q., Yuan, Y., Tang, X., Yang, Z., Zou, D., Bian, L. J., DAI, L. J., & LI, D. S. (2016) · Oncology Letters · doi:10.3892/ol.2016.4297 · Cited by 28
The mRNA vaccine, a swift warhead against a moving infectious disease target
Mir, S., & Mir, M. A. (2024) · Expert Review of Vaccines · doi:10.1080/14760584.2024.2320327 · Cited by 28
mRNA cancer vaccines from bench to bedside: a new era in cancer immunotherapy
Shariati, A., Khani, P., Nasri, F., Afkhami, H., Khezrpour, A., Kamrani, S., Shariati, F. S., Alavimanesh, S., & Modarressi, M. H. (2024) · Biomarker Research · doi:10.1186/s40364-024-00692-9 · Cited by 27
The Yin and Yang of nucleic acid-based therapy in the brain
Gustincich, S., Zucchelli, S., & Mallamaci, A. (2016) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2016.11.001 · Cited by 27
Khalil, A. S., Hellenbrand, D. J., Reichl, K., Umhoefer, J. M., Filipp, M. E., Choe, J. A., Hanna, A. S., & Murphy, W. L. (2022) · Advanced Healthcare Materials · doi:10.1002/adhm.202200206 · Cited by 27
Aligned Nanofibrillar Scaffolds for Controlled Delivery of Modified mRNA
Zaitseva, T. S., Alcazar, C. A., Zamani, M., Hou, L., Sawamura, S. J., Yakubov, E., Hopkins, M., Woo, Y.-P. J., Paukshto, M. V., & Huang, N. F. (2018) · Tissue Engineering Part A · doi:10.1089/ten.tea.2017.0494 · Cited by 27
M 3 RNA Drives Targeted Gene Delivery in Acute Myocardial Infarction
Singh, R. D., Hillestad, M. L., Livia, C., Li, M., Alekseev, A. E., Witt, T. A., Stalboerger, P. G., Yamada, S., Terzic, A., & Behfar, A. (2018) · Tissue Engineering Part A · doi:10.1089/ten.tea.2017.0445 · Cited by 27
(Dis)Solving the problem of aberrant protein states
Fare, C. M., & Shorter, J. (2021) · Disease Models & Mechanisms · doi:10.1242/dmm.048983 · Cited by 27
mRNAid, an open-source platform for therapeutic mRNA design and optimization strategies
Vostrosablin, N., Lim, S., Gopal, P., Brazdilova, K., Parajuli, S., Wei, X., Gromek, A., Příhoda, D., Spale, M., Muždalo, A., et al. (2024) · NAR Genomics and Bioinformatics · doi:10.1093/nargab/lqae028 · Cited by 30
Bates, S. M., Munson, M. J., Trovisco, V., Pereira, S. G. T., Miller, S. R., Sabirsh, A., Betts, C. J., Blenke, E. O., & Gay, N. J. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.114047 · Cited by 26
Uta Griesenbach, D., Davies, J. C., & Alton, E. W. F. W. (2016) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000000327 · Cited by 26
Large Animal Models of Cell-Free Cardiac Regeneration
Spannbauer, A., Mester-Tonczar, J., Traxler, D., Kastner, N., Zlabinger, K., Hašimbegović, E., Riesenhuber, M., Pávó, N., Goliasch, G., & Gyöngyösi, M. (2020) · Biomolecules · doi:10.3390/biom10101392 · Cited by 26
Delivery of self-replicating messenger RNA into the brain for the treatment of ischemic stroke
Kim, M., Oh, J., Lee, Y., Lee, E.-H., Ko, S. H., Jeong, J. H., Park, C., & Lee, M. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.08.049 · Cited by 26
mRNA-Mediated Gene Supplementation of Toll-Like Receptors as Treatment Strategy for Asthma In Vivo
Zeyer, F., Mothes, B., Will, C., Carevic, M., Rottenberger, J., Nürnberg, B., Hartl, D., Handgretinger, R., Beer‐Hammer, S., & Kormann, M. (2016) · PLoS ONE · doi:10.1371/journal.pone.0154001 · Cited by 27
Self-citationRomito, M., Juillerat, A., Kok, Y. L., Hildenbeutel, M., Rhiel, M., Andrieux, G., Geiger, J. P., Rudolph, C., Mussolino, C., Duclert, A., et al. (2020) · Biotechnology Journal · doi:10.1002/biot.202000023 · Cited by 27
Self-citationStrategies for improving the genome-editing efficiency of class 2 CRISPR/Cas system
Wang, L., & Han, H. (2024) · Heliyon · doi:10.1016/j.heliyon.2024.e38588 · Cited by 26
Synthesis of Modified mRNA for Myocardial Delivery
Kondrat, J., Sultana, N., & Zangi, L. (2016) · Methods in molecular biology · doi:10.1007/978-1-4939-6588-5_8 · Cited by 25
Tendon healing induced by chemically modified mRNAs
Groth, K., Berezhanskyy, T., Aneja, M., Geiger, J. P., Schweizer, M., Maucksch, L., Pasewald, T., Brill, T., Tigani, B., Weber, E., et al. (2017) · European Cells and Materials · doi:10.22203/ecm.v033a22 · Cited by 25
Self-citationNucleic acid therapies for cystic fibrosis
Sasaki, S., & Guo, S. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0696 · Cited by 24
Biologics for bone regeneration: advances in cell, protein, gene, and mRNA therapies
Runzer, C. D. T., Balmayor, E. R., & van Griensven, M. (2026) · Bone Research · doi:10.1038/s41413-025-00487-0 · Cited by 24
Loomis, K. H., Kirschman, J. L., Bhosle, S. M., Bellamkonda, R. V., & Santangelo, P. J. (2015) · Journal of Materials Chemistry B · doi:10.1039/c5tb01753j · Cited by 24
Selection and Identification of Skeletal-Muscle-Targeted RNA Aptamers
Philippou, S., Mastroyiannopoulos, N. P., Makrides, N., Lederer, C. W., Kleanthous, M., & Phylactou, L. A. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.12.004 · Cited by 24
Elwakil, M. M. A., Gao, T., Isono, T., Sato, Y., Elewa, Y. H. A., Satoh, T., & Harashima, H. (2021) · Materials Horizons · doi:10.1039/d1mh00185j · Cited by 24
Fleming, A. M., & Burrows, C. J. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad044 · Cited by 25
Wolfson, D. W., Kim, N. K., Lee, K. H., Beyersdorf, J. P., Langberg, J. J., Fernandez, N., Choi, D., Zureick, N., Kim, T. Y., Bae, S., et al. (2024) · Nature Biomedical Engineering · doi:10.1038/s41551-024-01211-9 · Cited by 24
Hansson, M. L., Albert, S., Somermeyer, L. G., Peco, R., Mejía-Ramírez, E., Montserrat, N., & Belmonte, J. C. I. (2015) · Journal of Biological Chemistry · doi:10.1074/jbc.m114.618835 · Cited by 25
Expert opinion in biological therapy: update on developments in lung gene transfer
Uta Griesenbach, D., & Alton, E. W. F. W. (2013) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2013.735656 · Cited by 25
Therapeutic editing of hepatocyte genome in vivo
de Galarreta, M. R., & Lujambio, A. (2017) · Journal of Hepatology · doi:10.1016/j.jhep.2017.05.012 · Cited by 25
Site-Specific Covalent Conjugation of Modified mRNA by tRNA Guanine Transglycosylase
Ehret, F., Zhou, C. Y., Alexander, S. C., Zhang, D., & Devaraj, N. K. (2017) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.7b00356 · Cited by 24
Akita, H. (2020) · Biological and Pharmaceutical Bulletin · doi:10.1248/bpb.b20-00534 · Cited by 23
Adverse immunological responses against non-viral nanoparticle (NP) delivery systems in the lung
de Braganca, L., Ferguson, G. J., Santos, J. L., & Derrick, J. P. (2021) · Journal of Immunotoxicology · doi:10.1080/1547691x.2021.1902432 · Cited by 24
mRNA delivery technologies: Toward clinical translation
Gómez-Aguado, I., Rodríguez-Castejón, J., Beraza-Millor, M., Rodríguez‐Gascón, A., del Pozo‐Rodríguez, A., & Solinís, M. Á. (2022) · International review of cytology · doi:10.1016/bs.ircmb.2022.04.010 · Cited by 23
De, A., & Ko, Y. T. (2022) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2023.2153832 · Cited by 24
Modified mRNA Therapeutics for Heart Diseases
Magadum, A. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232415514 · Cited by 23
Lipidoid mRNA Nanoparticles for Myocardial Delivery in Rodents
Turnbull, I. C., Eltoukhy, A. A., Anderson, D. G., & Costa, K. D. (2016) · Methods in molecular biology · doi:10.1007/978-1-4939-6588-5_10 · Cited by 23
Kurimoto, S., Yoshinaga, N., Igarashi, K., Matsumoto, Y., Cabral, H., & Uchida, S. (2019) · Molecules · doi:10.3390/molecules24071303 · Cited by 23
Munis, A. M., Hyde, S. C., & Gill, D. R. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.11.013 · Cited by 23
Direct Reprogramming—The Future of Cardiac Regeneration?
Doppler, S. A., Deutsch, M.-A., Lange, R., & Krane, M. (2015) · International Journal of Molecular Sciences · doi:10.3390/ijms160817368 · Cited by 23
Co-Delivery of mRNA and pDNA Using Thermally Stabilized Coacervate-Based Core-Shell Nanosystems
Nasr, S. S., Lee, S., Thiyagarajan, D., Boese, A. S., Loretz, B., & Lehr, C. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13111924 · Cited by 22
Rapid Delivery of Nanobodies/VHHs into Living Cells via Expressing In Vitro-Transcribed mRNA
Zhou, X., Hao, R., Chen, C., Su, Z., Zhao, L., Luo, Z., & Xie, W. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.01.008 · Cited by 22
Multivalent Peptide-Functionalized Bioreducible Polymers for Cellular Delivery of Various RNAs
Yang, D., Eldredge, A. C., Hickey, J. C., Muradyan, H., & Guan, Z. (2020) · Biomacromolecules · doi:10.1021/acs.biomac.0c00211 · Cited by 22
Fayed, O., van Griensven, M., Birgani, Z. N. T., Plank, C., & Balmayor, E. R. (2021) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c01042 · Cited by 22
van Asbeck, A. H., Dieker, J. W. C., Egberink, R. O., van den Berg, L., van der Vlag, J., & Brock, R. E. (2021) · Biomedicines · doi:10.3390/biomedicines9050511 · Cited by 21
Nuclear-Import Receptors Counter Deleterious Phase Transitions in Neurodegenerative Disease
Odeh, H. M., Fare, C. M., & Shorter, J. (2021) · Journal of Molecular Biology · doi:10.1016/j.jmb.2021.167220 · Cited by 22
The Potential of Nanomedicine to Unlock the Limitless Applications of mRNA
Taina-González, L., & de la Fuente, M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14020460 · Cited by 23
mRNA-based therapies: Preclinical and clinical applications
Qureischi, M., Mohr, J., Arellano-Viera, E., Knudsen, S. E., Vohidov, F., & Garitano-Trojaola, A. (2022) · International review of cytology · doi:10.1016/bs.ircmb.2022.04.007 · Cited by 21
Peletta, A., Lemoine, C., Courant, T., Collin, N., & Borchard, G. (2023) · Pharmacological Research · doi:10.1016/j.phrs.2023.106699 · Cited by 21
Detection of EPO gene doping in blood
Neuberger, E. W. I., Jurkiewicz, M., Moser, D., & Simon, P. D. (2012) · Drug Testing and Analysis · doi:10.1002/dta.1347 · Cited by 22
Kukreja, A., Lasaro, M. A., Cobaugh, C., Forbes, C. D., Tang, J., Gao, X., Martín-Higueras, C., Pey, A. L., SALIDO, E. C., Sobolov, S. B., et al. (2019) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0740 · Cited by 21
Legere, R. M., Cohen, N. D., Poveda, C., Bray, J. M., Barhoumi, R., Szule, J. A., de la Concha-Bermejillo, A., Bordin, A. I., & Pollet, J. (2021) · Scientific Reports · doi:10.1038/s41598-020-79855-1 · Cited by 20
Tam, Y. K., Madden, T. D., & Hope, M. J. (2016) · Journal of Drug Targeting · doi:10.1080/1061186x.2016.1221955 · Cited by 20
Material Design for Next-Generation mRNA Vaccines Using Lipid Nanoparticles
Higuchi, A., Sung, T., Wang, T., Ling, Q., Kumar, S. S., Hsu, S.-T., & Umezawa, A. (2022) · Journal of macromolecular science. Part C, Reviews in macromolecular chemistry and physics/Journal of macromolecular science. Reviews in macromolecular chemistry and physics · doi:10.1080/15583724.2022.2106490 · Cited by 21
Cafri, G., Sharbi‐Yunger, A., Tzehoval, E., Alteber, Z., Gross, T., Vadai, E., Margalit, A., Gross, G., & Eisenbach, L. (2015) · Molecular Therapy · doi:10.1038/mt.2015.90 · Cited by 20
Barnett, R. C., Lin, X., Barravecchia, M., Norman, R., Bentley, K. L. D. M., Fazal, F., Young, J. L., & Dean, D. A. (2017) · Experimental Biology and Medicine · doi:10.1177/1535370217713000 · Cited by 20
Michel, T., Golombek, S., Steinle, H., Hann, L., Velić, A., Maček, B., Krajewski, S., Schlensak, C., Wendel, H. P., & Avci‐Adali, M. (2019) · Journal of Biological Engineering · doi:10.1186/s13036-019-0172-5 · Cited by 21
mRNA produced by VSW-3 RNAP has high-level translation efficiency with low inflammatory stimulation
Wang, G., Cheng, R., Chen, Q., Xu, Y., Yu, B., Zhu, B., Yin, H., & Xia, H. (2022) · Cell Insight · doi:10.1016/j.cellin.2022.100056 · Cited by 20
Yu, X., Liu, S., Cheng, Q., Lee, S. M., Wei, T., Zhang, D., Farbiak, L., Johnson, L. T., Wang, X., & Siegwart, D. J. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13111914 · Cited by 20
IgA vasculitis following COVID-19 vaccination
Nishimura, N., SHIOMICHI, Y., Takeuchi, S., Akamine, S., Yoneda, R., & Yoshizawa, S. (2022) · Modern Rheumatology Case Reports · doi:10.1093/mrcr/rxac014 · Cited by 19
Runzer, C. D. T., Anand, S., Mota, C., Moroni, L., Plank, C., van Griensven, M., & Balmayor, E. R. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.05.019 · Cited by 19
Ely, A., Singh, P., Smith, T. S., & Arbuthnot, P. B. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.05.010 · Cited by 19
Optimal Chimeric Antigen Receptor (CAR)-mRNA for Transient CAR T Cell Generation
Kitte, R., Serfling, R., Blache, U., Seitz, C., Schrader, S., Koehl, U., Fricke, S., Bär, C., & Tretbar, S. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26030965 · Cited by 18
Non-Viral RNA Therapies for Non-Small Cell Lung Cancer and Their Corresponding Clinical Trials
Tiwade, P. B., Fung, V., VanKeulen‐Miller, R., Narasipura, E. A., Ma, Y., & Fenton, O. S. (2025) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.4c00871 · Cited by 18
Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs
Fenton, O. S., Kauffman, K. J., McClellan, R. L., Kaczmarek, J. C., Zeng, M. D., Andresen, J. L., Rhym, L. H., Heartlein, M. W., DeRosa, F., & Anderson, D. G. (2018) · Angewandte Chemie · doi:10.1002/ange.201809056 · Cited by 18
Non-Viral Delivery of Gene Therapy to the Tendon
Jin, J., Yang, Q., & Zhou, Y. L. (2022) · Polymers · doi:10.3390/polym14163338 · Cited by 18
Translating cancer research by synthetic biology
Shankar, S., & Pillai, M. R. (2011) · Molecular BioSystems · doi:10.1039/c1mb05016h · Cited by 18
Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies
Antony, J. S., Dewerth, A., Haque, A., Handgretinger, R., & Kormann, M. (2015) · Molecular and Cellular Pediatrics · doi:10.1186/s40348-015-0022-6 · Cited by 18
Self-citationAbraham, M.-K., Nolte, A., Reus, R., Behring, A., Zengerle, D., Avci‐Adali, M., Hohmann, J. D., Peter, K., Schlensak, C., Wendel, H. P., et al. (2015) · PLoS ONE · doi:10.1371/journal.pone.0138375 · Cited by 18
Reprogramming cell fate with artificial transcription factors
Heiderscheit, E. A., Eguchi, A., Spurgat, M. C., & Ansari, A. Z. (2018) · FEBS Letters · doi:10.1002/1873-3468.12993 · Cited by 18
Transgene-free direct conversion of murine fibroblasts into functional muscle stem cells
Qabrati, X., Kim, I., Ghosh, A., Bundschuh, N., Noé, F., Palmer, A. S., & Bar‐Nur, O. (2023) · npj Regenerative Medicine · doi:10.1038/s41536-023-00317-z · Cited by 18
Wang, X. L., Hu, P., Guo, X., Ding, Y., Yuan, Y., Yan, S. R., & Li, D. S. (2014) · Cytotherapy · doi:10.1016/j.jcyt.2014.05.017 · Cited by 17
Single-cell kinetics of siRNA-mediated mRNA degradation
Krzysztoń, R., Woschée, D., Reiser, A., Schwake, G., Strey, H. H., & Rädler, J. O. (2019) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2019.102077 · Cited by 20
RNA-Based Strategies for Cell Reprogramming toward Pluripotency
Bailly, A., Milhavet, O., & Lemaı̂tre, J.-M. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14020317 · Cited by 18
Alameh, M., & Weissman, D. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-821595-1.00014-2 · Cited by 17
LINE-1 RNA triggers matrix formation in bone cells via a PKR-mediated inflammatory response
Mangiavacchi, A., Morelli, G., Reppe, S., Saera-Vila, A., Liu, P., Eggerschwiler, B., Zhang, H., Bensaddek, D., Casanova, E. A., Medina‐Gómez, C., et al. (2024) · The EMBO Journal · doi:10.1038/s44318-024-00143-z · Cited by 16
mRNA vaccine development and applications: A special focus on tumors (Review)
Chen, B., Yang, Y., Wang, X., Yang, W., Lu, Y., Wang, D., Zhuo, E., Tang, Y., Su, J., Tang, G., et al. (2024) · International Journal of Oncology · doi:10.3892/ijo.2024.5669 · Cited by 17
Mokuda, S., Watanabe, H., Kohno, H., Ishitoku, M., Araki, K., Hirata, S., & Sugiyama, E. (2022) · Cytotechnology · doi:10.1007/s10616-022-00540-4 · Cited by 16
Solodushko, V., & Fouty, B. (2023) · Gene Therapy · doi:10.1038/s41434-023-00391-4 · Cited by 18
Quaternization drives spleen-to-lung tropism conversion for mRNA-loaded lipid-like nanoassemblies
Huang, Y., Wu, J., Li, S., Liu, Z., Li, Z., Zhou, B., & Li, B. (2024) · Theranostics · doi:10.7150/thno.90071 · Cited by 16
Exploring the role of pericardial miRNAs and exosomes in modulating cardiac fibrosis
Schoettler, F. I., Hassanabad, A. F., Jadli, A. S., Patel, V. B., & Fedak, P. W. M. (2024) · Cardiovascular Pathology · doi:10.1016/j.carpath.2024.107671 · Cited by 16
Gene Therapy: The View from NCATS
Brooks, P. J., Yang, N. N., & Austin, C. P. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.29018.pjb · Cited by 16
Messenger RNA as a Novel Therapeutic Approach
Stanton, M. G., & Murphy-Benenato, K. E. (2017) · Topics in medicinal chemistry · doi:10.1007/7355_2016_30 · Cited by 16
Therapeutic Mechanism of Nucleic Acid Drugs
Liu, L., Gao, H., Guo, C., Liu, T., Li, N., & Qian, Q. (2021) · ChemistrySelect · doi:10.1002/slct.202002901 · Cited by 16
Giordano, L., Aneja, M. K., Sommer, N., Alebrahimdehkordi, N., Saraji, A. A., Weißmann, N., Rudolph, C., Plank, C., Jacobs, H. T., & Szibor, M. (2021) · Gene Therapy · doi:10.1038/s41434-021-00235-z · Cited by 16
Self-citationYoshinaga, N., Naito, M., Tachihara, Y., Boonstra, E., Osada, K., Cabral, H., & Uchida, S. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13060800 · Cited by 16
In Vitro Evaluation of a Nanoparticle-Based mRNA Delivery System for Cells in the Joint
Sturm, L., Faustini, B., Menzel, U., Häckel, S., Albers, C. E., Plank, C., Rip, J., Alini, M., Traweger, A., Grad, S., et al. (2021) · Biomedicines · doi:10.3390/biomedicines9070794 · Cited by 16
Lee, K., Kim, T., Seo, Y., Kim, S. Y., & Lee, H. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.08.009 · Cited by 16
Khan, M., Musazzi, U. M., Manellari, S., Mouawad, N., Damiano, G., Rinaldi, R., Raucci, A., Costantino, S., Paneni, F., Minghetti, P., et al. (2025) · Pharmacological Research · doi:10.1016/j.phrs.2025.107847 · Cited by 16
Casmil, I. C., Friesen, J. J., Bathula, N. V., Strumpel, A., Ho, C.-H., Guez, I., Kong, K. Y. S., Varley, A., Miyake-Stoner, S. J., Aliahmad, P., et al. (2025) · Advanced Science · doi:10.1002/advs.202508907 · Cited by 17
Modern Strategies of Drug Therapy: Multi-Target Drug Delivery, Bioimaging, Diagnostics
Fedorova, O. V., Ovchinnikova, I. G., Rusinov, G. L., Avdeeva, V. V., Zhdanov, А. P., Zhizhin, K. Y., Kuznetsov, N. T., Zakharova, L. Y., Kuznetsova, D. A., Razuvayeva, Y. S., et al. (2025) · Russian Journal of General Chemistry · doi:10.1134/s1070363225606726 · Cited by 16
Rosenecker, J., Guan, S., Darmstädter, M., & Xu, C. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13081281 · Cited by 15
Self-citationRNA Engineering for Public Health: Innovations in RNA-Based Diagnostics and Therapeutics
Thavarajah, W., Hertz, L. M., Bushhouse, D. Z., Archuleta, C. M., & Lucks, J. B. (2021) · Annual Review of Chemical and Biomolecular Engineering · doi:10.1146/annurev-chembioeng-101420-014055 · Cited by 15
The potential application and challenge of powerful CRISPR/Cas9 system in cardiovascular research
Li, Y., Song, Y., Liu, B., & Yu, X. (2016) · International Journal of Cardiology · doi:10.1016/j.ijcard.2016.11.177 · Cited by 15
Metadata
- Authors
- Kormann, M. S. D., Hasenpusch, G., Aneja, M. K., Nica, G., Flemmer, A. W., Herber-Jonat, S., Huppmann, M., Mays, L. E., Illenyi, M., Schams, A., Griese, M., Bittmann, I., Handgretinger, R., Hartl, D., Rosenecker, J., & Rudolph, C.
- Affiliations
- Ludwig-Maximilians-Universität München (DE)
- University Children's Hospital Tübingen (DE)
- Agaplesion Diakonieklinikum Rotenburg (DE)
- Corresponding author
- Kormann, Michael S D
- Rudolph, Carsten
- ORCID
- Venue
- Nature Biotechnology
- Published
- 2011-01-09
- Volume
- 29
- Issue
- 2
- Pages
- 154-157
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- OpenAlex
- W2071302752
- Cited by
- 733
- References
- 27
- Citations by year
- 2026: 26 · 2025: 41 · 2024: 59 · 2023: 54 · 2022: 64 · 2021: 61 · 2020: 69 · 2019: 53 · 2018: 56 · 2017: 58 · 2016: 65 · 2015: 48 · 2014: 23 · 2013: 28 · 2012: 17
- FWCI
- 10.12
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
- Keywords
- chemically modified mRNA, mRNA therapeutics, erythropoietin, surfactant protein B, immunogenicity, Toll-like receptors, RIG-I, intramuscular injection, hematocrit, non-viral gene therapy, lung disease
- MeSH terms
- Animals, Erythropoietin, Histocytochemistry, Lung, Mice, Transgenic, Proteolipids, RNA, Messenger, Gene Transfer Techniques, RNA Stability, Mice, Kaplan-Meier Estimate
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
