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.
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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
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Items 501–600 of 731
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Hay, B. A., Li, J., & Guo, M. (2017) · Theriogenology · doi:10.1016/j.theriogenology.2017.11.003 · Cited by 15
Rescue of STAT3 Function in Hyper-IgE Syndrome Using Adenine Base Editing
Eberherr, A. C., Maaske, A., Wolf, C., Giesert, F., Berutti, R., Rusha, E., Pertek, A., Kastlmeier, M. T., Voss, C., Plummer, M. D., et al. (2021) · The CRISPR Journal · doi:10.1089/crispr.2020.0111 · Cited by 15
Cell Engineering with Synthetic Messenger RNA
Rabinovich, P. M., & Weissman, S. M. (2012) · Methods in molecular biology · doi:10.1007/978-1-62703-260-5_1 · Cited by 15
Efficient ex vivo delivery of chemically modified messenger RNA using lipofection and magnetofection
Badieyan, Z. S., Pasewald, T., Mykhaylyk, O. M., Rudolph, C., & Plank, C. (2016) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2016.11.113 · Cited by 14
Self-citationUnlocking the potential of RNA-based therapeutics in the lung: current status and future directions
Man, H. S. J., Moosa, V. A., Singh, A. N., Wu, L., Granton, J. T., Juvet, S., Hoang, C. D., & de Perrot, M. E. (2023) · Frontiers in Genetics · doi:10.3389/fgene.2023.1281538 · Cited by 14
Najafi, Z., Rahmanian‐Devin, P., Rahimi, V. B., Nokhodchi, A., & Askari, V. R. (2024) · Fundamental and Clinical Pharmacology · doi:10.1111/fcp.12999 · Cited by 14
Advances in Development of mRNA-Based Therapeutics
Huang, L., Zhang, L., Li, W., Li, S., Wen, J., Li, H., & Liu, Z. (2020) · Current topics in microbiology and immunology · doi:10.1007/82_2020_222 · Cited by 16
Zalai, D., Kopp, J., Kozma, B., Küchler, M., Herwig, C., & Kager, J. (2020) · Drug Discovery Today : Technologies · doi:10.1016/j.ddtec.2021.04.001 · Cited by 13
Michel, T., Kankura, A., Medina, M. L. S., Kurz, J., Behring, A., Avci‐Adali, M., Nolte, A., Schlensak, C., Wendel, H. P., & Krajewski, S. (2015) · Nucleic Acid Therapeutics · doi:10.1089/nat.2015.0537 · Cited by 14
Corritore, E., Lee, Y.-S., Pasquale, V., Liberati, D., Hsu, M., Lombard, C. A., Smissen, P. V. D., Vetere, A., Bonner‐Weir, S., Piemonti, L., et al. (2016) · Stem Cells Translational Medicine · doi:10.5966/sctm.2015-0318 · Cited by 14
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Burrows, C. J., & Fleming, A. M. (2023) · Accounts of Chemical Research · doi:10.1021/acs.accounts.3c00458 · Cited by 13
Cruz-Pavlovich, F. J. S., Bolaños-Chang, A. J., Rio-Murillo, X. I. D., Aranda-Preciado, G. A., Razura-Ruiz, E. M., Santos, A., & Navarro‐Partida, J. (2024) · Cells · doi:10.3390/cells13020179 · Cited by 13
Non-viral delivery of RNA for therapeutic T cell engineering
Berdecka, D., Smedt, S. C. D., Vos, W. H. D., & Braeckmans, K. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115215 · Cited by 14
Wang, S., Guo, B., Wang, H., & Yang, F. (2024) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2024.105547 · Cited by 14
Wei, Z., Zhang, S., Wang, X., Xue, Y., Dang, S., & Zhai, J. (2025) · Frontiers in Immunology · doi:10.3389/fimmu.2025.1524317 · Cited by 16
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Zhang, Y., Xi, X., Yu, H., Yang, L., Lin, J., Yang, W., Liu, J., Fan, X., & Xu, Y. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.08.017 · Cited by 13
Hausburg, F., Na, S., Voronina, N., Skorska, A., Müller, P., Steinhoff, G., & David, R. (2015) · Cellular Physiology and Biochemistry · doi:10.1159/000373957 · Cited by 13
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Mahaling, B., Roy, C., & Ghosh, S. (2024) · Journal of Materials Chemistry B · doi:10.1039/d4tb00491d · Cited by 12
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Ting, J., Tamayo-Mendoza, T., Petersen, S. R., Reet, J. V., Ahmed, U. A., Snell, N. J., Fisher, J. D., Stern, M., & Oviedo, F. (2023) · Chemical Communications · doi:10.1039/d3cc04705a · Cited by 12
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Hirschberger, K., Jarzębińska, A., Kessel, E., Kretzschmann, V. K., Aneja, M. K., Dohmen, C., Herrmann-Janson, A., Wagner, E., Plank, C., & Rudolph, C. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.12.006 · Cited by 12
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Liu, Y., Liu, X., Huang, J., Shi, Y., Luo, Z., Zhang, J., Guo, X., Jiang, M., Li, X., Yin, H., et al. (2022) · Advanced Healthcare Materials · doi:10.1002/adhm.202202460 · Cited by 11
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Azimi, S. M., Sheridan, S. D., Ghannad‐Rezaie, M., Eimon, P. M., & Yanik, M. F. (2018) · eLife · doi:10.7554/elife.31922 · Cited by 11
Oswald, A., Chakraborty, A., Ni, Y., Wettengel, J. M., Urban, S., & Protzer, U. (2021) · Scientific Reports · doi:10.1038/s41598-021-99263-3 · Cited by 11
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Lui, K. O., Bu, L., Li, R. A., & Chan, C. W. Y. (2012) · Birth Defects Research Part C Embryo Today Reviews · doi:10.1002/bdrc.21004 · Cited by 10
Wang, J., Endsley, A. N., Green, C. E., & Matin, A. (2016) · BMC Cancer · doi:10.1186/s12885-016-2508-6 · Cited by 10
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Oldenhuis, N. J., Eldredge, A. C., Burts, A. O., Ryu, K. A., Chung, J., Johnson, M. E., & Guan, Z. (2016) · ChemistrySelect · doi:10.1002/slct.201600939 · Cited by 10
Nagaraj, S., Stankiewicz-Drogoń, A., Darżynkiewicz, E., & Grzela, R. (2022) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2022.1017934 · Cited by 9
Elovic, E., Etzion, S., & Cohen, S. (2019) · Scientific Reports · doi:10.1038/s41598-019-50899-2 · Cited by 9
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Macht, A., Huang, Y., Reinert, L. S., Grass, V., Lohmer, K., Prada, E. T. A., Babel, E., Semmler, A., Zhang, W., Wegner, A., et al. (2024) · EMBO Reports · doi:10.1038/s44319-024-00216-4 · Cited by 8
Self-citationLü, T., Chen, A., Li, C., Li, K., Wang, S., Zhang, Y., Yang, B., Wang, J., Gong, Q., Li, A., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf963 · Cited by 8
Jarzębińska, A., Pasewald, T., Lambrecht, J., Mykhaylyk, O. M., Kümmerling, L., Beck, P., Hasenpusch, G., Rudolph, C., Plank, C., & Dohmen, C. (2016) · Angewandte Chemie · doi:10.1002/ange.201603648 · Cited by 8
Self-citationVincenzo, F. D., Ennas, S., Pizzoferrato, M., Bibbò, S., Porcari, S., Ianiro, G., & Cammarota, G. (2023) · Internal and Emergency Medicine · doi:10.1007/s11739-023-03366-w · Cited by 8
Antony, J. S., Birrer, P., Bohnert, C., Zimmerli, S. E., Hillmann, P., Schaffhauser, H., Hoeflich, C., Hoeflich, A., Khairallah, R. J., Satoh, A. T., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.102055 · Cited by 8
Bush, A. (2023) · Paediatric Respiratory Reviews · doi:10.1016/j.prrv.2023.11.001 · Cited by 8
Leontovyč, I., Habart, D., Loukotová, Š., Košinová, L., Kříž, J., Saudek, F., & Koblas, T. (2017) · PLoS ONE · doi:10.1371/journal.pone.0182497 · Cited by 8
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Suscovich, T. J., & Alter, G. (2015) · Expert Review of Vaccines · doi:10.1586/14760584.2015.1001375 · Cited by 9
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Duvall, C. L., Prokop, A., Gersbach, C. A., & Davidson, J. M. (2013) · Elsevier eBooks · doi:10.1016/b978-0-12-398358-9.00035-5 · Cited by 8
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Torrisi, S. A., Geraci, F., Diolosà, L., Luca, A. D., Falzone, L., Drago, F., Libra, M., & Leggio, G. M. (2025) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2025.108958 · Cited by 9
Miliotou, Α. N., Foltopoulou, P. F., Ingendoh‐Tsakmakidis, A., Tsiftsoglou, A. S., Vizirianakis, I. S., Pappas, I. S., & Papadopoulou, L. C. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15010286 · Cited by 7
McCaughey‐Chapman, A., Burgers, A. L., Combrinck, C., Marriott, L., Gordon, D., & Connor, B. J. (2024) · Stem Cell Research & Therapy · doi:10.1186/s13287-024-04057-9 · Cited by 7
Gupta, R., & Mohanty, S. (2016) · Materials Science and Engineering C · doi:10.1016/j.msec.2016.09.012 · Cited by 7
Michel, T., Link, A., Abraham, M.-K., Schlensak, C., Peter, K., Wendel, H.-P., Wang, X., & Krajewski, S. (2019) · Journal of Visualized Experiments · doi:10.3791/58444 · Cited by 7
Zaitseva, T. S., Yang, G., Dionyssiou, D. D., Zamani, M., Sawamura, S. J., Yakubov, E., Ferguson, J., Hallett, R. L., Fleischmann, D., Paukshto, M. V., et al. (2020) · Regenerative Medicine · doi:10.2217/rme-2020-0023 · Cited by 7
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Slagter-Jäger, J. G., Raney, A., Lewis, W. E., Debenedette, M. A., Nicolette, C. A., & Tcherepanova, I. Y. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.18 · Cited by 7
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Huleihel, L., Scarritt, M. E., & Badylak, S. F. (2017) · Current Pathobiology Reports · doi:10.1007/s40139-017-0121-2 · Cited by 7
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Zeng, Y., Zhao, G., Wu, S., Bochuan, H., Forn‐Cuní, G., Knol, R. A., Ghalbzouri, A. E., Snaar-Jagalska, E. B., & Kros, A. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.114598 · Cited by 6
Pawar, S., Pingale, P., Garkal, A., Osmani, R. A. M., Gajbhiye, K. R., Kulkarni, M., Pardeshi, K., Mehta, T. A., & Rajput, A. (2024) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2024.131139 · Cited by 6
Faustini, B., Lettner, T., Wagner, A., Tempfer, H., Cesur, N. P., Lehner, C., Brouwer, C., Roelofs, K., Mykhailyk, O., Plank, C., et al. (2025) · Acta Biomaterialia · doi:10.1016/j.actbio.2025.02.025 · Cited by 7
Recent strategies for enhanced delivery of mRNA to the lungs
Heiser, B. J., Veyssi, A., & Ghosh, D. (2025) · Nanomedicine · doi:10.1080/17435889.2025.2485669 · Cited by 7
Harnessing cell reprogramming for cardiac biological pacing
Liu, C., Chen, Y., & Hu, Y. (2023) · Journal of Biomedical Science · doi:10.1186/s12929-023-00970-y · Cited by 6
Next-Generation Therapeutics: mRNA as a Novel Therapeutic Option for Single-Gene Disorders
Michel, T., Wendel, H.-P., & Krajewski, S. (2016) · InTech eBooks · doi:10.5772/62243 · Cited by 7
Transgene Site-Specific Integration: Problems and Solutions
Bire, S., & Rouleux‐Bonnin, F. (2012) · Topics in current genetics · doi:10.1007/978-94-007-4531-5_1 · Cited by 6
mRNA-Based Nanomedicine: A New Strategy for Treating Infectious Diseases and Beyond
Prakash, S. (2023) · European Journal of Drug Metabolism and Pharmacokinetics · doi:10.1007/s13318-023-00849-1 · Cited by 6
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Lin, P. J. C., & Tam, Y. K. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-814470-1.00009-5 · Cited by 6
Xiao, Y., Xu, H., Guo, W. Q., Zhao, Y., Luo, Y., Wang, M., He, Z., Ding, Z., Liu, J., Deng, L., et al. (2020) · Molecular Biomedicine · doi:10.1186/s43556-020-00017-w · Cited by 6
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Nelson, C. E., Duvall, C. L., Prokop, A., Gersbach, C. A., & Davidson, J. M. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-818422-6.00030-7 · Cited by 6
Hori, M., Qiao, N., Yamada, K., Yum, J., Ogura, S., Uchida, S., Kim, H. J., Naito, M., & Miyata, K. (2026) · ACS Applied Bio Materials · doi:10.1021/acsabm.6c00050 · Cited by 5
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Srivastava, A., Chauhan, S., & Ahuja, V. (2023) · Karbala International Journal of Modern Science · doi:10.33640/2405-609x.3330 · Cited by 6
A non-lipid nucleic acid delivery vector with dendritic cell tropism and stimulation
Kim, H. S., Halabi, E. A., Enbergs, N., Köhler, R. H., Fei, F., Garris, C., & Weissleder, R. (2024) · Theranostics · doi:10.7150/thno.95267 · Cited by 5
Oligonucleotide therapeutics in sports? An antidoping perspective
Parr, M. K., & Keiler, A. M. (2024) · Archiv der Pharmazie · doi:10.1002/ardp.202400404 · Cited by 5
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Rozema, D. B. (2017) · Annual reports in medicinal chemistry · doi:10.1016/bs.armc.2017.07.003 · Cited by 5
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Yılmaz, E. (2021) · Mikrobiyoloji Bulteni · doi:10.5578/mb.20219912 · Cited by 7
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Dhanjal, D. S., Singh, R., Sharma, V., Nepovimová, E., Adam, V., Kuča, K., & Chopra, C. (2023) · Current Medicinal Chemistry · doi:10.2174/0929867330666230503144619 · Cited by 5
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Tan, F., Du, Z., Zhong, J., Wu, Y., Mou, J., Zhao, F., Liu, Y., Chen, J., Liang, Z., Zhou, Y., et al. (2024) · Current Medicinal Chemistry · doi:10.2174/0109298673277325231229093344 · Cited by 5
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Cai, J., Chen, S., Liu, Z., Li, H., Wang, P., Yang, F., Li, Y., Chen, K., Sun, M., & Qiu, M. (2024) · Immunology · doi:10.1111/imm.13861 · Cited by 5
Green, D. W., Kim, E., & Jung, H.-S. (2015) · Journal of Biomedical Materials Research Part A · doi:10.1002/jbm.a.35414 · Cited by 5
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Fernández, E. F., Bangel‐Ruland, N., Tomczak, K., & Weber, W.-M. (2016) · Translational Medicine Communications · doi:10.1186/s41231-016-0006-0 · Cited by 5
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Weng, Y., & Huang, Y. (2019) · Springer eBooks · doi:10.1007/978-981-13-8731-9_7 · Cited by 5
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Papadopol, M. D. (2009) · Americanae (AECID Library) · Cited by 5
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Xue, D., Zhang, Y., Shen, A., Tang, T., Liu, B., & Lv, L. (2025) · Interdisciplinary medicine · doi:10.1002/inmd.20240054 · Cited by 5
Li, M., Zubair, A., Wdidi, S., & He, S. (2026) · Journal of Cellular and Molecular Medicine · doi:10.1111/jcmm.70986 · Cited by 4
Feng, Z., Chu, L., Li, Q., Zhou, J., Wu, P., Zhang, X., Pan, Y., Zou, J., Chen, Q., Xu, Z., et al. (2026) · Nature Communications · doi:10.1038/s41467-026-68364-w · Cited by 4
Yu, X., Deng, X.-M., Lin, Y., Ren, H., Jia, L., Meng, Y., Liu, F., Cheng, Q., Teng, Z., & Wei, T. (2026) · ACS Nano · doi:10.1021/acsnano.6c04580 · Cited by 5
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
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