Nanoscale platforms for messenger RNA delivery

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

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Endocytosis of nanomedicines

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

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Exploring polyethylenimine‐mediated DNA transfection and the proton sponge hypothesis

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A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

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Lipid Nanoparticle Systems for Enabling Gene Therapies

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Codon Optimality Is a Major Determinant of mRNA Stability

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Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes

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

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Cationic liposome-mediated RNA transfection.

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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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Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

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

Finn, J. D., Smith, A. R., Patel, M. C., Shaw, L., Youniss, M. R., van Heteren, J., Dirstine, T., Ciullo, C., Lescarbeau, R., Seitzer, J., et al. (2018) · Cell Reports · doi:10.1016/j.celrep.2018.02.014 · Cited by 878 · see citing works

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

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

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

Oberli, M. A., Reichmuth, A. M., Dorkin, J. R., Mitchell, M. J., Fenton, O. S., Jaklenec, A., Anderson, D. G., Langer, R., & Blankschtein, D. (2017) · Nano Letters · doi:10.1021/acs.nanolett.6b03329 · Cited by 719 · see citing works

The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery

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Nonviral delivery of self-amplifying RNA vaccines

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Regulation of cytoplasmic mRNA decay

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PEG — A versatile conjugating ligand for drugs and drug delivery systems

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Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

Whitehead, K. A., Dorkin, J. R., Vegas, A. J., Chang, P. H., Veiseh, O., Matthews, J., Fenton, O. S., Zhang, Y., Olejnik, K. T., Yesilyurt, V., et al. (2014) · Nature Communications · doi:10.1038/ncomms5277 · Cited by 615 · see citing works

CRISPR/Cas9-based genome editing for disease modeling and therapy: challenges and opportunities for nonviral delivery

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The systemic toxicity of positively charged lipid nanoparticles and the role of Toll-like receptor 4 in immune activation

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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

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Delivery of Therapeutic Proteins

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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

Induction of virus‐specific cytotoxic T lymphocytes <i>in vivo</i> by liposome‐entrapped mRNA

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Protamine sulfate enhances lipid-mediated gene transfer

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The Centrality of RNA

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Dendrimer-RNA nanoparticles generate protective immunity against lethal Ebola, H1N1 influenza, and <i>Toxoplasma gondii</i> challenges with a single dose

Chahal, J. S., Khan, O. F., Cooper, C. L., McPartlan, J. S., Tsosie, J. K., Tilley, L. D., Sidik, S. M., Lourido, S., Langer, R., Bavari, S., et al. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1600299113 · Cited by 414 · see citing works

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 400 · see citing works

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 · see citing works

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

Weide, B., Pascolo, S., Scheel, B., Derhovanessian, E., Pflugfelder, A., Eigentler, T. K., Pawelec, G., Hoerr, I., Rammensee, H.-G., & Garbe, C. (2009) · Journal of Immunotherapy · doi:10.1097/cji.0b013e3181a00068 · Cited by 378 · cited 2× · see citing works

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

Polymer–Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

Kaczmarek, J. C., Patel, A. K., Kauffman, K. J., Fenton, O. S., Webber, M. J., Heartlein, M. W., DeRosa, F., & Anderson, D. G. (2016) · Angewandte Chemie International Edition · doi:10.1002/anie.201608450 · Cited by 337 · see citing works

Cancer immunotherapy with mRNA‐transfected dendritic cells

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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 · see citing works

A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo

Jiang, C., Mei, M., Li, B., Zhu, X., Zu, W., Tian, Y., Wang, Q., Guo, Y., Dong, Y., & Tan, X. (2017) · Cell Research · doi:10.1038/cr.2017.16 · Cited by 295 · see citing works

Synthesis and Biological Evaluation of Ionizable Lipid Materials for the In Vivo Delivery of Messenger RNA to B Lymphocytes

Fenton, O. S., Kauffman, K. J., Kaczmarek, J. C., McClellan, R. L., Jhunjhunwala, S., Tibbitt, M. W., Zeng, M. D., Appel, E. A., Dorkin, J. R., Mir, F. F., et al. (2017) · Advanced Materials · doi:10.1002/adma.201606944 · Cited by 289 · see citing works

Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals

McKinlay, C. J., Vargas, J. R., Blake, T. R., Hardy, J. W., Kanada, M., Contag, C. H., Wender, P. A., & Waymouth, R. M. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1614193114 · Cited by 288 · see citing works

<i>In Vitro</i> and <i>in Vivo</i> mRNA Delivery Using Lipid-Enveloped pH-Responsive Polymer Nanoparticles

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An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo

Li, B., Luo, X., Deng, B., Wang, J., McComb, D. W., Shi, Y., Gaensler, K. M. L., Tan, X., Dunn, A. L., Kerlin, B. A., et al. (2015) · Nano Letters · doi:10.1021/acs.nanolett.5b03528 · Cited by 261 · see citing works

Chapter 17 Messenger RNA Half‐Life Measurements in Mammalian Cells

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Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery

Fenton, O. S., Kauffman, K. J., McClellan, R. L., Appel, E. A., Dorkin, J. R., Tibbitt, M. W., Heartlein, M. W., DeRosa, F., Langer, R., & Anderson, D. G. (2016) · Advanced Materials · doi:10.1002/adma.201505822 · Cited by 254 · see citing works

Boosting intracellular delivery of lipid nanoparticle-encapsulated mRNA

Patel, S., Ashwanikumar, N., Robinson, E., DuRoss, A., Sun, C., Murphy-Benenato, K. E., Mihai, C., Almarsson, Ö., & Sahay, G. (2017) · Nano Letters · doi:10.1021/acs.nanolett.7b02664 · Cited by 251 · see citing works

mRNA transcript therapy

Weissman, D. (2015) · Expert Review of Vaccines · doi:10.1586/14760584.2015.973859 · Cited by 228 · see citing works

Translation of rabbit globin mRNA introduced by liposomes into mouse lymphocytes

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Lipid-based mRNA vaccine delivery systems

Midoux, P., & Pichon, C. (2014) · Expert Review of Vaccines · doi:10.1586/14760584.2015.986104 · Cited by 209 · see citing works

Enhanced intranasal delivery of mRNA vaccine by overcoming the nasal epithelial barrier via intra- and paracellular pathways

Li, M., Zhao, M., Fu, Y., Li, Y., Gong, T., Zhang, Z., & Sun, X. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.02.043 · Cited by 201 · see citing works

Toll‐like receptor‐dependent activation of several human blood cell types by protamine‐condensed mRNA

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Safety Evaluation of Lipid Nanoparticle–Formulated Modified mRNA in the Sprague-Dawley Rat and Cynomolgus Monkey

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 · cited 3× · see citing works

Current prospects for mRNA gene delivery

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Systemic delivery of modified mRNA encoding herpes simplex virus 1 thymidine kinase for targeted cancer gene therapy.

Wang, Y.-H., Su, H.-H., Yang, Y., Hu, Y.-X., Zhang, L., Blancafort, P., & Huang, L. (2013) · Molecular Therapy · doi:10.1038/mt.2012.250 · Cited by 189 · see citing works

Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery

Alabi, C. A., Love, K. T., Sahay, G., Yin, H., Luly, K. M., Langer, R., & Anderson, D. G. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1306529110 · Cited by 188 · see citing works

Self-Amplifying mRNA Vaccines

Brito, L. A., Kommareddy, S., Maione, D., Uematsu, Y., Giovani, C., Scorza, F. B., Otten, G. R., Yu, D. L., Mandl, C. W., Mason, P. W., et al. (2014) · Advances in genetics · doi:10.1016/bs.adgen.2014.10.005 · Cited by 182 · see citing works

Potent Immune Responses in Rhesus Macaques Induced by Nonviral Delivery of a Self-amplifying RNA Vaccine Expressing HIV Type 1 Envelope With a Cationic Nanoemulsion

Bogers, W. M., Oostermeijer, H., Mooij, P., Koopman, G., Verschoor, E. J., Davis, D., Ulmer, J. B., Brito, L. A., Cu, Y., Banerjee, K., et al. (2015) · Journal of Infectious Diseases · doi:10.1093/infdis/jiu522 · Cited by 181 · see citing works

Biomaterials for mRNA delivery

Islam, M. A., Reesor, E. K. G., Xu, Y., Zope, H. R., Zetter, B. R., & Shi, J. (2015) · Biomaterials Science · doi:10.1039/c5bm00198f · Cited by 176 · see citing works

Arginine‐Rich Peptide‐Based mRNA Nanocomplexes Efficiently Instigate Cytotoxic T Cell Immunity Dependent on the Amphipathic Organization of the Peptide

Udhayakumar, V. K., De Beuckelaer, A., McCaffrey, J., McCrudden, C. M., Kirschman, J. L., Vanover, D., Van Hoecke, L., Roose, K., Deswarte, K., De Geest, B. G., et al. (2017) · Advanced Healthcare Materials · doi:10.1002/adhm.201601412 · Cited by 169 · cited 3× · see citing works

An RNA nanoparticle vaccine against Zika virus elicits antibody and CD8+ T cell responses in a mouse model

Chahal, J. S., Fang, T., Woodham, A. W., Khan, O. F., Ling, J. J., Anderson, D. G., & Ploegh, H. L. (2017) · Scientific Reports · doi:10.1038/s41598-017-00193-w · Cited by 161 · see citing works

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

DeRosa, F., Guild, B., Karve, S., Smith, L., Love, K., Dorkin, J. R., Kauffman, K. J., Zhang, J., Yahalom, B., Anderson, D. G., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.46 · Cited by 159 · see citing works

Effects of Chemically Modified Messenger RNA on Protein Expression

Li, B., Luo, X. L., & Dong, Y. (2016) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.6b00090 · Cited by 154 · see citing works

Delivering the right message: Challenges and opportunities in lipid nanoparticles-mediated modified mRNA therapeutics—An innate immune system standpoint

Granot-Matok, Y., & Peer, D. (2017) · Seminars in Immunology · doi:10.1016/j.smim.2017.08.015 · Cited by 154 · see citing works

Systemic delivery of messenger RNA for the treatment of pancreatic cancer using polyplex nanomicelles with a cholesterol moiety

Uchida, S., Kinoh, H., Ishii, T., Matsui, A., Tockary, T. A., Takeda, K. M., Uchida, H., Osada, K., Itaka, K., & Kataoka, K. (2015) · Biomaterials · doi:10.1016/j.biomaterials.2015.12.031 · Cited by 150 · see citing works

Chitosan/Hyaluronic Acid Nanoparticles: Rational Design Revisited for RNA Delivery

Lallana, E., Rosa, J. M. R. D. L., Tirella, A., Pelliccia, M., Gennari, A., Stratford, I. J., Puri, S., Ashford, M. B., & Tirelli, N. (2017) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.7b00320 · Cited by 146 · see citing works

Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia

Nabhan, J. F., Wood, K. M., Rao, V. P., Morin, J., Bhamidipaty, S., LaBranche, T. P., Gooch, R. L., Bozal, F., Bulawa, C. E., & Guild, B. C. (2016) · Scientific Reports · doi:10.1038/srep20019 · Cited by 140 · see citing works

Self-replicating alphavirus RNA vaccines

Ljungberg, K., & Liljeström, P. (2014) · Expert Review of Vaccines · doi:10.1586/14760584.2015.965690 · Cited by 137 · see citing works

Lipid-Based Nanoparticles in The Systemic Delivery of siRNA

Lin, Q., Chen, J., Zhang, Z., & Zheng, G. (2013) · Nanomedicine · doi:10.2217/nnm.13.192 · Cited by 133 · see citing works

Induction of HIV-1 gag specific immune responses by cationic micelles mediated delivery of gag mRNA

Zhao, M., Li, M., Zhang, Z., Gong, T., & Sun, X. (2016) · Drug Delivery · doi:10.3109/10717544.2015.1038856 · Cited by 132 · see citing works

Co-delivery of nucleoside-modified mRNA and TLR agonists for cancer immunotherapy: restoring the immunogenicity of immunosilent mRNA

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Multifunctional triblock copolymers for intracellular messenger RNA delivery

Cheng, C., Convertine, A. J., Stayton, P. S., & Bryers, J. D. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.06.020 · Cited by 123 · see citing works

Efficient mRNA delivery with graphene oxide-polyethylenimine for generation of footprint-free human induced pluripotent stem cells

Choi, H. Y., Lee, T., Yang, G., Oh, J., Won, J., Han, J., Jeong, G., Kim, J., Kim, J., Kim, B., et al. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.06.007 · Cited by 114 · see citing works

Evading innate immunity in nonviral mRNA delivery: don't shoot the messenger.

Devoldere, J., Dewitte, H., De Smedt, S. C., & Remaut, K. (2016) · Drug Discovery Today · doi:10.1016/j.drudis.2015.07.009 · Cited by 112 · see citing works

Messenger RNA delivery of a cartilage-anabolic transcription factor as a disease-modifying strategy for osteoarthritis treatment

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Myocardial Delivery of Lipidoid Nanoparticle Carrying modRNA Induces Rapid and Transient Expression

Turnbull, I. C., Eltoukhy, A. A., Fish, K. M., Nonnenmacher, M. E., Ishikawa, K., Chen, J., Hajjar, R. J., Anderson, D. G., & Costa, K. D. (2015) · Molecular Therapy · doi:10.1038/mt.2015.193 · Cited by 112 · see citing works

Translating Nanoparticulate-Personalized Cancer Vaccines Into Clinical Applications: Case Study With RNA-Lipoplexes for the Treatment of Melanoma

Grabbe, S., Haas, H., Diken, M., Kranz, L. M., Langguth, P., & Şahin, U. (2016) · Nanomedicine · doi:10.2217/nnm-2016-0275 · Cited by 112 · see citing works

Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo

Dong, Y., Dorkin, J. R., Wang, W., Chang, P., Webber, M. J., Tang, B. C., Yang, J., Ionita, I. A., Danino, D., DeRosa, F., et al. (2016) · Nano Letters · doi:10.1021/acs.nanolett.5b02428 · Cited by 111 · see citing works

Systemic mRNA delivery to the lungs by functional polyester-based carriers

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Polyethylenimine-based polyplex delivery of self-replicating RNA vaccines

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Biodegradable Amino-Ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo

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