The challenge and prospect of mRNA therapeutics landscape

Exosome-mediated transfer of mRNAs and microRNAs is a novel mechanism of genetic exchange between cells

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A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine.

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Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure.

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mRNA vaccines — a new era in vaccinology

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Direct Gene Transfer into Mouse Muscle in Vivo

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Species-specific recognition of single-stranded RNA via toll-like receptor 7 and 8

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Innate antiviral responses by means of TLR7-mediated recognition of single-stranded RNA

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Regulation of Translation Initiation in Eukaryotes: Mechanisms and Biological Targets

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Suppression of RNA Recognition by Toll-like Receptors: The Impact of Nucleoside Modification and the Evolutionary Origin of RNA

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Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA

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mRNA-based therapeutics--developing a new class of drugs

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5'-Triphosphate RNA Is the Ligand for RIG-I

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Cancer immunotherapy via dendritic cells

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Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

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Innate immune recognition of viral infection

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Immune Signaling by RIG-I-like Receptors

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Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy

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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

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Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

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An Unstable Intermediate Carrying Information from Genes to Ribosomes for Protein Synthesis

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Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

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Selected Toll-like receptor agonist combinations synergistically trigger a T helper type 1–polarizing program in dendritic cells

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Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

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Lipid-like materials for low-dose, in vivo gene silencing

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Optimization of lipid nanoparticles for intramuscular administration of mRNA vaccines

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Dendritic cells pulsed with RNA are potent antigen-presenting cells in vitro and in vivo.

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Cationic liposome-mediated RNA transfection.

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RNA-Based Therapeutics: Current Progress and Future Prospects

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Modified mRNA vaccines protect against zika virus infection

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Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

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A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

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 · see citing works

The cap and poly(A) tail function synergistically to regulate mRNA translational efficiency.

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RNA-Targeted Therapeutics

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A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

Sabnis, S., Kumarasinghe, E. S., Salerno, T., Mihai, C., Ketova, T., Senn, J. J., Lynn, A., Bulychev, A., McFadyen, I., Chan, J., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.03.010 · Cited by 820 · see citing works

Tools for translation: non-viral materials for therapeutic mRNA delivery

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Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

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 785 · see citing works

Large-scale generation of functional mRNA-encapsulating exosomes via cellular nanoporation

Yang, Z., Shi, J., Xie, J., Wang, Y., Sun, J., Liu, T., Zhao, Y., Zhao, X., Wang, X., Ma, Y., et al. (2019) · Nature Biomedical Engineering · doi:10.1038/s41551-019-0485-1 · Cited by 744 · see citing works

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

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Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

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Clinical use of dendritic cells for cancer therapy

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N1-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice

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Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

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Nonviral delivery of self-amplifying RNA vaccines

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Efficient RNA drug delivery using red blood cell extracellular vesicles

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Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

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Preclinical and clinical demonstration of immunogenicity by mRNA vaccines against H10N8 and H7N9 influenza viruses

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Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

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Modification of antigen-encoding RNA increases stability, translational efficacy, and T-cell stimulatory capacity of dendritic cells

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Functional Specializations of Human Epidermal Langerhans Cells and CD14+ Dermal Dendritic Cells

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In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

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Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

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Induction of CD8+T-Cell Responses Against Novel Glioma–Associated Antigen Peptides and Clinical Activity by Vaccinations With α-Type 1 Polarized Dendritic Cells and Polyinosinic-Polycytidylic Acid Stabilized by Lysine and Carboxymethylcellulose in Patients With Recurrent Malignant Glioma

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Autologous dendritic cells transfected with prostate-specific antigen RNA stimulate CTL responses against metastatic prostate tumors

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mRNA structure regulates protein expression through changes in functional half-life

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Non‐Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co‐Delivery of Cas9 mRNA and sgRNA

Miller, J. B., Zhang, S., Kos, P., Xiong, H., Zhou, K., Perelman, S. S., Zhu, H., & Siegwart, D. J. (2017) · Angewandte Chemie International Edition · doi:10.1002/anie.201610209 · Cited by 540 · see citing works

Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles

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Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals

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 · see citing works

A comparison of non-integrating reprogramming methods

Schlaeger, T. M., Daheron, L. M., Brickler, T., Entwisle, S. W., Chan, K., Cianci, A. L., DeVine, A. L., Ettenger, A., Fitzgerald, K. N., Godfrey, M., et al. (2014) · Nature Biotechnology · doi:10.1038/nbt.3070 · Cited by 512 · see citing works

An RNA vaccine drives expansion and efficacy of claudin-CAR-T cells against solid tumors

Reinhard, K., Rengstl, B., Oehm, P., Michel, K., Billmeier, A., Hayduk, N., Klein, O., Kuna, K., Ouchan, Y., Wöll, S., et al. (2020) · Science · doi:10.1126/science.aay5967 · Cited by 512 · see citing works

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

Yin, H., Song, C.-Q., Suresh, S., Wu, Q., Walsh, S., Rhym, L. H., Mintzer, E., Bolukbasi, M. F., Zhu, L. J., Kauffman, K., et al. (2017) · Nature Biotechnology · doi:10.1038/nbt.4005 · Cited by 499 · see citing works

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

Dong, Y., Love, K. T., Dorkin, J. R., Sirirungruang, S., Zhang, Y., Chen, D., Bogorad, R. L., Yin, H., Chen, Y., Vegas, A. J., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1322937111 · Cited by 488 · see citing works

Autologous dendritic cells transfected with prostate-specific antigen RNA stimulate CTL responses against metastatic prostate tumors

Heiser, A., Coleman, D., Dannull, J., Yancey, D. R., Maurice, M. A., Lallas, C. D., Dahm, P., Niedzwiecki, D., Gilboa, E., & Vieweg, J. (2002) · Journal of Clinical Investigation · doi:10.1172/jci14364 · Cited by 487 · see citing works

Highly efficient gene delivery by mRNA electroporation in human hematopoietic cells: superiority to lipofection and passive pulsing of mRNA and to electroporation of plasmid cDNA for tumor antigen loading of dendritic cells

Van Tendeloo, V. F. I., Ponsaerts, P., Lardon, F., Nijs, G., Lenjou, M., Van Broeckhoven, C., Van Bockstaele, D. R., & Berneman, Z. N. (2001) · Blood · doi:10.1182/blood.v98.1.49 · Cited by 468 · see citing works

Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection

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TAIL-seq: Genome-wide Determination of Poly(A) Tail Length and 3′ End Modifications

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Antiviral Signaling Through Pattern Recognition Receptors

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Multiple Injections of Electroporated Autologous T Cells Expressing a Chimeric Antigen Receptor Mediate Regression of Human Disseminated Tumor

Zhao, Y., Moon, E. K., Carpenito, C., Paulos, C. M., Liu, X., Brennan, A. L., Chew, A., Carroll, R. G., Scholler, J., Levine, B. L., et al. (2010) · Cancer Research · doi:10.1158/0008-5472.can-10-2880 · Cited by 448 · see citing works

Immunotherapy of cancer in 2012

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Dendritic cell maturation: functional specialization through signaling specificity and transcriptional programming

Dalod, M., Chelbi, R., MALISSEN, B., & Lawrence, T. (2014) · The EMBO Journal · doi:10.1002/embj.201488027 · Cited by 430 · see citing works

RIG-I and Other RNA Sensors in Antiviral Immunity

Chow, K. T., Gale, M., & Loo, Y. (2018) · Annual Review of Immunology · doi:10.1146/annurev-immunol-042617-053309 · Cited by 429 · see citing works

Generation of mutant mice by pronuclear injection of circular plasmid expressing Cas9 and single guided RNA

Mashiko, D., Fujihara, Y., Satouh, Y., Miyata, H., Isotani, A., & Ikawa, M. (2013) · Scientific Reports · doi:10.1038/srep03355 · Cited by 426 · see citing works

Genomewide characterization of non-polyadenylated RNAs

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Enhanced Gene Delivery and siRNA Silencing by Gold Nanoparticles Coated with Charge-Reversal Polyelectrolyte

Guo, S., Huang, Y., Jiang, Q., et al. (2010) · ACS Nano · doi:10.1021/nn101638u · Cited by 403 · see citing works

Efficient Targeting and Activation of Antigen-Presenting Cells In Vivo after Modified mRNA Vaccine Administration in Rhesus Macaques

Liang, F., Lindgren, G., Lin, A., Thompson, E. A., Ols, S., Röhss, J., John, S., Hassett, K., Yuzhakov, O., Bahl, K., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.08.006 · Cited by 395 · see citing works

Inhaled 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 · see citing works

Direct Injection of Protamine-protected mRNA: Results of a Phase 1/2 Vaccination Trial in Metastatic Melanoma Patients

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Purification of mRNA guanylyltransferase and mRNA (guanine-7-) methyltransferase from vaccinia virions.

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Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge

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 · see citing works

The role of liposomes in clinical nanomedicine development. What now? Now what?

Crommelin, D. J. A., van Hoogevest, P., & Storm, G. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.12.023 · Cited by 350 · see citing works

Speeding with control: codon usage, tRNAs, and ribosomes

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A Cationic Nanoemulsion for the Delivery of Next-generation RNA Vaccines

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Genetically Engineered Microvesicles Carrying Suicide mRNA/Protein Inhibit Schwannoma Tumor Growth

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