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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Lipid nanoparticles for mRNA delivery
Hou, X., Zaks, T., Langer, R., & Dong, Y. (2021) · Nature Reviews Materials · doi:10.1038/s41578-021-00358-0 · Cited by 4,076
Non-viral vectors for gene-based therapy
Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123
mRNA-based therapeutics--developing a new class of drugs
Sahin, U., Karikó, K., & Türeci, Ö. (2014) · Nature Reviews Drug Discovery · doi:10.1038/nrd4278 · Cited by 2,474
Therapeutic siRNA: state of the art
Hu, B., Zhong, L., Weng, Y., Peng, L., Huang, Y., Zhao, Y., & Liang, X.-J. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-020-0207-x · Cited by 1,586
The evolution of commercial drug delivery technologies
Vargason, A. M., Anselmo, A. C., & Mitragotri, S. (2021) · Nature Biomedical Engineering · doi:10.1038/s41551-021-00698-w · Cited by 1,361
Therapeutic genome editing: prospects and challenges
Cox, D. B. T., Platt, R. J., & Zhang, F. (2015) · Nature Medicine · doi:10.1038/nm.3793 · Cited by 1,336
Block copolymer micelles in nanomedicine applications
Cabral, H., Miyata, K., Osada, K., & Kataoka, K. (2018) · Chemical Reviews · doi:10.1021/acs.chemrev.8b00199 · Cited by 1,309
Overcoming cellular barriers for RNA therapeutics
Dowdy, S. F. (2017) · Nature Biotechnology · doi:10.1038/nbt.3802 · Cited by 1,139
Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery
Kowalski, P. S., Rudra, A., Miao, L., & Anderson, D. G. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.012 · Cited by 1,127
Rethinking cancer nanotheranostics
Chen, H., Zhang, W., Zhu, G., Xie, J., & Chen, X. S. (2017) · Nature Reviews Materials · doi:10.1038/natrevmats.2017.24 · Cited by 1,113
Smart nanoparticles for cancer therapy
Sun, L., Liu, H., Ye, Y., Lei, Y., Islam, R., Tan, S., Tong, R., Miao, Y.-B., & Cai, L. (2023) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-023-01642-x · Cited by 1,073
Emerging Frontiers in Drug Delivery
Tibbitt, M. W., Dahlman, J. E., & Langer, R. (2016) · Journal of the American Chemical Society · doi:10.1021/jacs.5b09974 · Cited by 1,005
Karikó, K., Muramatsu, H., Ludwig, J., & Weissman, D. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr695 · Cited by 898
mRNA capping: biological functions and applications
Ramanathan, A., Robb, G. B., & Chan, S. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw551 · Cited by 961
Yin, H., Song, C.-Q., Dorkin, J. R., Zhu, L. J., Li, Y., Wu, Q., Park, A., Yang, J., Suresh, S., Bizhanova, A., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3471 · Cited by 876
Advances in Biomaterials for Drug Delivery
Fenton, O. S., Olafson, K. N., Pillai, P. S., Mitchell, M. J., & Langer, R. (2018) · Advanced Materials · doi:10.1002/adma.201705328 · Cited by 888
The delivery of therapeutic oligonucleotides
Juliano, R. L. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw236 · Cited by 878
Tools for translation: non-viral materials for therapeutic mRNA delivery
Hajj, K. A., & Whitehead, K. A. (2017) · Nature Reviews Materials · doi:10.1038/natrevmats.2017.56 · Cited by 799
Kauffman, K. J., Dorkin, J. R., Yang, J. H., Heartlein, M. W., DeRosa, F., Mir, F. F., Fenton, O. S., & Anderson, D. G. (2015) · Nano Letters · doi:10.1021/acs.nanolett.5b02497 · Cited by 796
Intracellular Delivery by Membrane Disruption: Mechanisms, Strategies, and Concepts
Stewart, M. P., Langer, R., & Jensen, K. F. (2018) · Chemical Reviews · doi:10.1021/acs.chemrev.7b00678 · Cited by 762
Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA
Long, C., McAnally, J. R., Shelton, J. M., Mireault, A. A., Bassel-Duby, R., & Olson, E. N. (2014) · Science · doi:10.1126/science.1254445 · Cited by 720
Andries, O., Mc Cafferty, S., De Smedt, S. C., Weiss, R., Sanders, N. N., & Kitada, T. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.08.051 · Cited by 701
Advances in mRNA Vaccines for Infectious Diseases
Zhang, C., Maruggi, G., Shan, H., & Li, J. (2019) · Frontiers in Immunology · doi:10.3389/fimmu.2019.00594 · Cited by 696
Advances in the delivery of RNA therapeutics: from concept to clinical reality
Kaczmarek, J. C., Kowalski, P. S., & Anderson, D. G. (2017) · Genome Medicine · doi:10.1186/s13073-017-0450-0 · Cited by 656
Zangi, L., Lui, K. O., von Gise, A., Ma, Q., Ebina, W., Ptaszek, L. M., Später, D., Xu, H., Tabebordbar, M., Gorbatov, R., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2682 · Cited by 646
Developing mRNA-vaccine technologies
Schlake, T., Thess, A., Fotin-Mleczek, M., & Kallen, K.-J. (2012) · RNA Biology · doi:10.4161/rna.22269 · Cited by 622
Unlocking the promise of mRNA therapeutics
Rohner, E., Yang, R., Foo, K. S., Goedel, A., & Chien, K. R. (2022) · Nature Biotechnology · doi:10.1038/s41587-022-01491-z · Cited by 612
Advances in COVID-19 mRNA vaccine development
Fang, E., Liu, X., Li, M., Zhang, Z., Song, L., Zhu, B., Wu, X., Liu, J., Zhao, D., & Li, Y. (2022) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-00950-y · Cited by 601
Nucleic Acid Therapeutics Using Polyplexes: A Journey of 50 Years (and Beyond)
Lächelt, U., & Wagner, E. (2015) · Chemical Reviews · doi:10.1021/cr5006793 · Cited by 607
Lipid nanoparticles for nucleic acid delivery: Current perspectives
Samaridou, E., Heyes, J. A., & Lutwyche, P. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.06.002 · Cited by 609
Opportunities and Challenges in the Delivery of mRNA-Based Vaccines
Wadhwa, A., Aljabbari, A., Lokras, A., Foged, C., & Thakur, A. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12020102 · Cited by 584
Wang, H.-X., Li, M., Lee, C. M., Chakraborty, S., Kim, H.-W., Bao, G., & Leong, K. W. (2017) · Chemical Reviews · doi:10.1021/acs.chemrev.6b00799 · Cited by 560
mRNA structure regulates protein expression through changes in functional half-life
Mauger, D. M., Cabral, B. J., Presnyak, V., Su, S. V., Reid, D. W., Goodman, B., Link, K., Khatwani, N., Reynders, J., Moore, M. J., et al. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1908052116 · Cited by 555
Yanez Arteta, M., Kjellman, T., Bartesaghi, S., Wallin, S., Wu, X., Kvist, A. J., Dabkowska, A., Székely, N., Radulescu, A., Bergenholtz, J., et al. (2018) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1720542115 · Cited by 534
Genetic programming of macrophages to perform anti-tumor functions using targeted mRNA nanocarriers
Zhang, F., Parayath, N. N., Ene, C., Stephan, S. B., Koehne, A. L., Coon, M. E., Holland, E. C., & Stephan, M. T. (2019) · Nature Communications · doi:10.1038/s41467-019-11911-5 · Cited by 527
Kim, J., Eygeris, Y., Gupta, M., & Sahay, G. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.12.014 · Cited by 514
Thess, A., Grund, S., Mui, B. L., Hope, M. J., Baumhof, P., Fotin-Mleczek, M., & Schlake, T. (2015) · Molecular Therapy · doi:10.1038/mt.2015.103 · Cited by 513
The landscape of mRNA nanomedicine
Huang, X., Kong, N., Zhang, X., Cao, Y., Langer, R., & Tao, W. (2022) · Nature Medicine · doi:10.1038/s41591-022-02061-1 · Cited by 511
Base editing: advances and therapeutic opportunities
Porto, E. M., Komor, A. C., Slaymaker, I. M., & Yeo, G. W.-M. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0084-6 · Cited by 495
Modifications in an Emergency: The Role of N1-Methylpseudouridine in COVID-19 Vaccines
Nance, K. D., & Meier, J. L. (2021) · ACS Central Science · doi:10.1021/acscentsci.1c00197 · Cited by 466
The Medicinal Chemistry of Therapeutic Oligonucleotides
Wan, W. B., & Seth, P. P. (2016) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.6b00551 · Cited by 445
The challenge and prospect of mRNA therapeutics landscape
Weng, Y., Li, C., Yang, T., Hu, B., Zhang, M., Guo, S., Xiao, H., Liang, X.-J., & Huang, Y. (2020) · Biotechnology Advances · doi:10.1016/j.biotechadv.2020.107534 · Cited by 417
Materials for non-viral intracellular delivery of messenger RNA therapeutics
Kauffman, K. J., Webber, M. J., & Anderson, D. G. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.12.032 · Cited by 406
Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems
Guan, S., & Rosenecker, J. (2017) · Gene Therapy · doi:10.1038/gt.2017.5 · Cited by 389
Self-citationInhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium
Patel, A. K., Kaczmarek, J. C., Bose, S., Kauffman, K. J., Mir, F. F., Heartlein, M. W., DeRosa, F., Langer, R., & Anderson, D. G. (2019) · Advanced Materials · doi:10.1002/adma.201805116 · Cited by 383
New Modalities for Challenging Targets in Drug Discovery
Valeur, E., Guéret, S. M., Adihou, H., Gopalakrishnan, R., Lemurell, M., Waldmann, H., Grossmann, T. N., & Plowright, A. T. (2017) · Angewandte Chemie International Edition · doi:10.1002/anie.201611914 · Cited by 393
Liu, L., Wang, Y., Miao, L., Liu, Q., Musetti, S., Li, J., & Huang, L. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.10.020 · Cited by 373
mRNA Vaccine Era—Mechanisms, Drug Platform and Clinical Prospection
Xu, S., Yang, K., Li, R., & Zhang, L. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21186582 · Cited by 368
Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems
Leung, A. K. K., Tam, Y. Y. C., Chen, S., Hafez, I. M., & Cullis, P. R. (2015) · The Journal of Physical Chemistry B · doi:10.1021/acs.jpcb.5b02891 · Cited by 359
Advances in Non-Viral DNA Vectors for Gene Therapy
Hardee, C. L., Arevalo-Soliz, L. M., Hornstein, B. D., & Zechiedrich, L. (2017) · Genes · doi:10.3390/genes8020065 · Cited by 369
RNA modifications in physiology and disease: towards clinical applications
Delaunay, S., Helm, M., & Frye, M. (2023) · Nature Reviews Genetics · doi:10.1038/s41576-023-00645-2 · Cited by 368
Impact of mRNA chemistry and manufacturing process on innate immune activation
Nelson, J., Sorensen, E. W., Mintri, S., Rabideau, A. E., Zheng, W., Besin, G., Khatwani, N., Su, S. V., Miracco, E. J., Issa, W. J., et al. (2020) · Science Advances · doi:10.1126/sciadv.aaz6893 · Cited by 354
In Vivo Gene Delivery by Nonviral Vectors: Overcoming Hurdles?
Zhang, Y., Satterlee, A., & Huang, L. (2012) · Molecular Therapy · doi:10.1038/mt.2012.79 · Cited by 361
Pardi, N., Secreto, A. J., Shan, X., Debonera, F., Glover, J., Yi, Y., Muramatsu, H., Ni, H., Mui, B. L., Tam, Y. K., et al. (2017) · Nature Communications · doi:10.1038/ncomms14630 · Cited by 352
Synergistic lipid compositions for albumin receptor mediated delivery of mRNA to the liver
Miao, L., Lin, J., Huang, Y., Li, L., Delcassian, D., Ge, Y., Shi, Y., & Anderson, D. G. (2020) · Nature Communications · doi:10.1038/s41467-020-16248-y · Cited by 343
Three decades of messenger RNA vaccine development
Verbeke, R., Lentacker, I., De Smedt, S. C., & Dewitte, H. (2019) · Nano Today · doi:10.1016/j.nantod.2019.100766 · Cited by 342
Islam, M. A., Xu, Y., Tao, W., Ubellacker, J. M., Lim, M., Aum, D., Lee, G. Y., Zhou, K., Zope, H., Yu, M., et al. (2018) · Nature Biomedical Engineering · doi:10.1038/s41551-018-0284-0 · Cited by 339
A Cationic Nanoemulsion for the Delivery of Next-generation RNA Vaccines
Brito, L. A., Chan, M., Shaw, C. A., Hekele, A., Carsillo, T., Schaefer, M., Archer, J., Seubert, A., Otten, G. R., Beard, C. W., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.133 · Cited by 338
RNA-based diagnostic and therapeutic strategies for cardiovascular disease
Lu, D., & Thum, T. (2019) · Nature Reviews Cardiology · doi:10.1038/s41569-019-0218-x · Cited by 341
Kong, N., Tao, W., Ling, X., Wang, J., Xiao, Y., Shi, S., Ji, X., Shajii, A., Gan, S. T., Kim, N. Y., et al. (2019) · Science Translational Medicine · doi:10.1126/scitranslmed.aaw1565 · Cited by 314
Polymers for siRNA Delivery: Inspired by Viruses to be Targeted, Dynamic, and Precise
Wagner, E. (2011) · Accounts of Chemical Research · doi:10.1021/ar2002232 · Cited by 309
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Karow, M., Sánchez, R., Schichor, C., Masserdotti, G., Ortega, F., Heinrich, C., Gascón, S., Khan, M. A., Lie, D. C., Dellavalle, A., et al. (2012) · Cell stem cell · doi:10.1016/j.stem.2012.07.007 · Cited by 316
Systemic delivery of factor IX messenger RNA for protein replacement therapy
Ramaswamy, S., Tonnu, N., Tachikawa, K., Limphong, P., Vega, J. B., Karmali, P. P., Chivukula, P., & Verma, I. M. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1619653114 · Cited by 301
A double-edged sword of immuno-microenvironment in cardiac homeostasis and injury repair
Sun, K., Li, Y., & Jin, J. (2021) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-020-00455-6 · Cited by 306
mRNA-based therapeutics: looking beyond COVID-19 vaccines
Parhiz, H., Atochina‐Vasserman, E. N., & Weissman, D. (2024) · The Lancet · doi:10.1016/s0140-6736(23)02444-3 · Cited by 303
Karikó, K., Muramatsu, H., Keller, J. M., & Weissman, D. (2012) · Molecular Therapy · doi:10.1038/mt.2012.7 · Cited by 282
Svitkin, Y. V., Cheng, Y. M., Chakraborty, T., Presnyak, V., John, M., & Sonenberg, N. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx135 · Cited by 278
Structure-function relationships in pulmonary surfactant membranes: From biophysics to therapy
López-Rodríguez, E., & Pérez‐Gil, J. (2014) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/j.bbamem.2014.01.028 · Cited by 276
Tunable protein synthesis by transcript isoforms in human cells
Floor, S. N., & Doudna, J. A. (2016) · eLife · doi:10.7554/elife.10921 · Cited by 265
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
An, D., Schneller, J. L., Frassetto, A., Liang, S., Zhu, X., Park, J.-S., Theisen, M., Hong, S.-J., Zhou, J., Rajendran, R., et al. (2017) · Cell Reports · doi:10.1016/j.celrep.2017.11.081 · Cited by 263
Vaidyanathan, S., Azizian, K. T., Haque, A. K. M. A., Henderson, J. M., Hendel, A., Shore, S., Antony, J. S., Hogrefe, R. I., Kormann, M. S. D., Porteus, M. H., et al. (2018) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2018.06.010 · Cited by 262
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CFTR: cystic fibrosis and beyond
Mall, M. A., & Hartl, D. (2014) · European Respiratory Journal · doi:10.1183/09031936.00228013 · Cited by 266
Self-citationKim, S. C., Sekhon, S. S., Shin, W.-R., Ahn, G., Cho, B.-K., Ahn, J.-Y., & Kim, Y.-H. (2022) · Molecular & Cellular Toxicology · doi:10.1007/s13273-021-00171-4 · Cited by 255
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Zhang, H., Zhang, Y., & Yin, H. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.01.014 · Cited by 255
In vivo gene delivery mediated by non-viral vectors for cancer therapy
Mohammadinejad, R., Dehshahri, A., Madamsetty, V. S., Zahmatkeshan, M., Tavakol, S., Makvandi, P., Khorsandi, D., Pardakhty, A., Ashrafizadeh, M., Afshar, E. G., et al. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.06.038 · Cited by 239
Strategies to reduce the risks of mRNA drug and vaccine toxicity
Bitounis, D., Jacquinet, E., Rogers, M. A., & Amiji, M. M. (2024) · Nature Reviews Drug Discovery · doi:10.1038/s41573-023-00859-3 · Cited by 242
An RNA toolbox for cancer immunotherapy
Pastor, F., Berraondo, P., Etxeberria, I., Frederick, J., Sahin, U., Gilboa, E., & Melero, I. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.132 · Cited by 229
Weissman, D. (2015) · Expert Review of Vaccines · doi:10.1586/14760584.2015.973859 · Cited by 228
Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives
Gómez-Aguado, I., Rodríguez-Castejón, J., Vicente-Pascual, M., Rodríguez‐Gascón, A., Solinís, M. Á., & del Pozo‐Rodríguez, A. (2020) · Nanomaterials · doi:10.3390/nano10020364 · Cited by 229
Cheng, Q., Wei, T., Jia, Y., Farbiak, L., Zhou, K., Zhang, S., Wei, Y., Zhu, H., & Siegwart, D. J. (2018) · Advanced Materials · doi:10.1002/adma.201805308 · Cited by 221
Efficient Detection and Purification of Cell Populations Using Synthetic MicroRNA Switches
Miki, K., Endo, K., Takahashi, S., Funakoshi, S., Takei, I., Katayama, S., Toyoda, T., Kotaka, M., Takaki, T., Umeda, M., et al. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.04.005 · Cited by 224
Reprogramming human fibroblasts to pluripotency using modified mRNA
Mandal, P., & Rossi, D. J. (2013) · Nature Protocols · doi:10.1038/nprot.2013.019 · Cited by 219
Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening
Guimarães, P. P. G., Zhang, R., Spektor, R., Tan, M., Chung, A., Billingsley, M. M., El-Mayta, R. D., Riley, R., Wang, L., Wilson, J. M., et al. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.10.028 · Cited by 224
Islam, M. A., Jamie, R., Reesor, E., Zope, H. R., Tao, W., Lim, M., Ding, J., Chen, Y., Aduluso, D., Zetter, B. R., et al. (2020) · Biomaterials · doi:10.1016/j.biomaterials.2020.120431 · Cited by 220
Hohwieler, M., Illing, A., Hermann, P. C., Mayer, T., Stockmann, M., Perkhofer, L., Eiseler, T., Antony, J. S., Müller, M., Renz, S., et al. (2016) · Gut · doi:10.1136/gutjnl-2016-312423 · Cited by 226
Self-citationLevy, O., Zhao, W., Mortensen, L. J., LeBlanc, S., Tsang, K., Fu, M., Phillips, J. A., Sagar, V., Anandakumaran, P. N., Ngai, J., et al. (2013) · Blood · doi:10.1182/blood-2013-04-495119 · Cited by 212
Non-viral COVID-19 vaccine delivery systems
Park, K. S., Sun, X., Aikins, M. E., & Moon, J. J. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.12.008 · Cited by 216
Naturally occurring modified ribonucleosides
McCown, P. J., Ruszkowska, A., Kunkler, C. N., Breger, K., Hulewicz, J. P., Wang, M. C., Springer, N. A., & Brown, J. A. (2020) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.1595 · Cited by 219
Kwon, H., Kim, M., Seo, Y., Moon, Y. S., Lee, H. J., Lee, K., & Lee, H. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2017.11.034 · Cited by 208
Wang, Y., Zhang, L., Xu, Z., Miao, L., & Huang, L. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.11.009 · Cited by 206
How mRNA therapeutics are entering the monoclonal antibody field
Van Hoecke, L., & Roose, K. (2019) · Journal of Translational Medicine · doi:10.1186/s12967-019-1804-8 · Cited by 205
In Vitro Transcription of Long RNA Containing Modified Nucleosides
Pardi, N., Muramatsu, H., Weissman, D., & Karikó, K. (2012) · Methods in molecular biology · doi:10.1007/978-1-62703-260-5_2 · Cited by 204
Drug and cell delivery for cardiac regeneration
Hastings, C. L., Roche, E. T., Ruiz‐Hernández, E., Schenke‐Layland, K., Walsh, C. J., & Duffy, G. P. (2014) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2014.08.006 · Cited by 205
Sedic, M., Senn, J. J., Lynn, A., Laska, M., Smith, M., Platz, S. J., Bolen, J., Hoge, S., Bulychev, A., Jacquinet, E., et al. (2018) · Veterinary Pathology · doi:10.1177/0300985817738095 · Cited by 195
Kauffman, K. J., Mir, F. F., Jhunjhunwala, S., Kaczmarek, J. C., Hurtado, J. E., Yang, J. H., Webber, M. J., Kowalski, P. S., Heartlein, M. W., DeRosa, F., et al. (2016) · Biomaterials · doi:10.1016/j.biomaterials.2016.09.006 · Cited by 195
RNA: The new revolution in nucleic acid vaccines
Geall, A. J., Mandl, C. W., & Ulmer, J. B. (2013) · Seminars in Immunology · doi:10.1016/j.smim.2013.05.001 · Cited by 192
Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination
Suberi, A., Grun, M. K., Mao, T., Israelow, B., Reschke, M., Grundler, J., Akhtar, L., Lee, T., Shin, K., Piotrowski-Daspit, A. S., et al. (2023) · Science Translational Medicine · doi:10.1126/scitranslmed.abq0603 · Cited by 190
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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