mRNA-based cancer therapeutics

A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

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Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

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RNA-Guided Human Genome Engineering via Cas9

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Efficient<i>in vitro</i>synthesis of biologically active RNA and RNA hybridization probes from plasmids containing a bacteriophage SP6 promoter

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Neoantigens in cancer immunotherapy

Schumacher, T. N., & Schreiber, R. D. (2015) · Science · doi:10.1126/science.aaa4971 · Cited by 4,783 · see citing works

mRNA vaccines — a new era in vaccinology

Pardi, N., Hogan, M. J., Porter, F. W., & Weissman, D. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2017.243 · Cited by 4,667 · see citing works

Direct Gene Transfer into Mouse Muscle in Vivo

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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 · see citing works

Poly(ethylene glycol) in Drug Delivery: Pros and Cons as Well as Potential Alternatives

Knop, K., Hoogenboom, R., Fischer, D., & Schubert, U. S. (2010) · Angewandte Chemie International Edition · doi:10.1002/anie.200902672 · Cited by 3,533 · see citing works

An immunogenic personal neoantigen vaccine for patients with melanoma

Ott, P. A., Hu, Z., Keskin, D. B., Shukla, S. A., Sun, J., Bozym, D. J., Zhang, W., Luoma, A., Giobbie-Hurder, A., Peter, L., et al. (2017) · Nature · doi:10.1038/nature22991 · Cited by 2,936 · see citing works

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

Karikó, K., Buckstein, M., Ni, H., & Weissman, D. (2005) · Immunity · doi:10.1016/j.immuni.2005.06.008 · Cited by 2,728 · see citing works

Progress and problems with the use of viral vectors for gene therapy

Thomas, C. E., Ehrhardt, A., & Kay, M. A. (2003) · Nature Reviews Genetics · doi:10.1038/nrg1066 · Cited by 2,501 · see citing works

Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer

Sahin, U., Derhovanessian, E., Miller, M., Kloke, B.-P., Simon, P., Löwer, M., Bukur, V., Tadmor, A. D., Luxemburger, U., Schrörs, B., et al. (2017) · Nature · doi:10.1038/nature23003 · Cited by 2,488 · see citing works

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 · see citing works

Senescence and tumour clearance is triggered by p53 restoration in murine liver carcinomas

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Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

Cheng, Q., Wei, T., Farbiak, L., Johnson, L. T., Dilliard, S. A., & Siegwart, D. J. (2020) · Nature Nanotechnology · doi:10.1038/s41565-020-0669-6 · Cited by 2,383 · see citing works

Toxicity of cationic lipids and cationic polymers in gene delivery

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Lipid NanoparticlesFrom Liposomes to mRNA Vaccine Delivery, a Landscape of Research Diversity and Advancement

Tenchov, R., Bird, R., Curtze, A. E., & Zhou, Q. (2021) · ACS Nano · doi:10.1021/acsnano.1c04996 · Cited by 2,221 · see citing works

Progress and challenges towards targeted delivery of cancer therapeutics

Rosenblum, D., Joshi, N., Tao, W., Karp, J. M., & Peer, D. (2018) · Nature Communications · doi:10.1038/s41467-018-03705-y · Cited by 2,220 · see citing works

Nanoparticle PEGylation for Imaging and Therapy

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Regulation of circRNA biogenesis

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Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection

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

Kranz, L. M., Diken, M., Haas, H., Kreiter, S., Loquai, C., Reuter, K. C., Meng, M., Fritz, D., Vascotto, F., Hefesha, H., et al. (2016) · Nature · doi:10.1038/nature18300 · Cited by 1,771 · see citing works

Cancer Immunotherapy Based on Mutation-Specific CD4+ T Cells in a Patient with Epithelial Cancer

Tran, E., Turcotte, S., Gros, A., Robbins, P. F., Lu, Y., Dudley, M. E., Wunderlich, J. R., Somerville, R. P. T., Hogan, K., Hinrichs, C. S., et al. (2014) · Science · doi:10.1126/science.1251102 · Cited by 1,710 · see citing works

mRNA vaccines for infectious diseases: principles, delivery and clinical translation

Chaudhary, N., Weissman, D., & Whitehead, K. A. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-021-00283-5 · Cited by 1,555 · see citing works

Loss of PTEN Promotes Resistance to T Cell–Mediated Immunotherapy

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Repair Pathway Choices and Consequences at the Double-Strand Break

Ceccaldi, R., Rondinelli, B., & D’Andrea, A. D. (2016) · Trends in Cell Biology · doi:10.1016/j.tcb.2015.07.009 · Cited by 1,499 · see citing works

CRISPR-engineered T cells in patients with refractory cancer

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The next generation of CRISPR–Cas technologies and applications

Pickar-Oliver, A., & Gersbach, C. A. (2019) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-019-0131-5 · Cited by 1,459 · see citing works

CAR T cells produced in vivo to treat cardiac injury

Rurik, J. G., Tombácz, I., Yadegari, A., Méndez Fernández, P. O., Shewale, S. V., Li, L., Kimura, T., Soliman, O. Y., Papp, T. E., Tam, Y. K., et al. (2022) · Science · doi:10.1126/science.abm0594 · Cited by 1,400 · see citing works

The current landscape of nucleic acid therapeutics

Kulkarni, J. A., Witzigmann, D., Thomson, S. B., Chen, S., Leavitt, B. R., Cullis, P. R., & van der Meel, R. (2021) · Nature Nanotechnology · doi:10.1038/s41565-021-00898-0 · Cited by 1,371 · see citing works

Mutant MHC class II epitopes drive therapeutic immune responses to cancer

Kreiter, S., Vormehr, M., van de Roemer, N., Diken, M., Löwer, M., Diekmann, J., Boegel, S., Schrörs, B., Vascotto, F., Castle, J. C., et al. (2015) · Nature · doi:10.1038/nature14426 · Cited by 1,243 · see citing works

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

Akinc, A., Zumbuehl, A., Goldberg, M. S., Leshchiner, E. S., Busini, V., Hossain, N., Bacallado, S., Nguyen, D. N., Fuller, J. E., Alvarez, R., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1402 · Cited by 1,228 · see citing works

Cytokines in clinical cancer immunotherapy

Berraondo, P., Sanmamed, M. F., Ochoa, M. C., Etxeberría, I., Aznar, M. A., Perez‐Gracia, J. L., Rodriguez-Ruiz, M. E., Ponz‐Sarvisé, M., Álvarez, E. C., & Melero, I. (2018) · British Journal of Cancer · doi:10.1038/s41416-018-0328-y · Cited by 1,190 · see citing works

Polymers for the sustained release of proteins and other macromolecules

LANGER, R., & FOLKMAN, J. (1976) · Nature · doi:10.1038/263797a0 · Cited by 1,182 · see citing works

Personalized vaccines for cancer immunotherapy

Şahin, U., & Türeci, Ö. (2018) · Science · doi:10.1126/science.aar7112 · Cited by 1,162 · see citing works

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 · see citing works

Targeting Strategies for Tissue-Specific Drug Delivery

Zhao, Z., Ukidve, A., Kim, J., & Mitragotri, S. (2020) · Cell · doi:10.1016/j.cell.2020.02.001 · Cited by 1,114 · see citing works

An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma

Sahin, U., Oehm, P., Derhovanessian, E., Jabulowsky, R. A., Vormehr, M., Gold, M., Maurus, D., Schwarck-Kokarakis, D., Kuhn, A. N., Omokoko, T., et al. (2020) · Nature · doi:10.1038/s41586-020-2537-9 · Cited by 1,052 · see citing works

Advances in the development of personalized neoantigen-based therapeutic cancer vaccines

Blass, E., & Ott, P. A. (2021) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-020-00460-2 · Cited by 1,042 · see citing works

Principles of Tumor Suppression

Sherr, C. J. (2004) · Cell · doi:10.1016/s0092-8674(03)01075-4 · Cited by 1,028 · see citing works

Mechanism and regulation of human non-homologous DNA end-joining

Lieber, M. R., Ma, Y., Pannicke, U., & Schwarz, K. (2003) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm1202 · Cited by 973 · see citing works

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

Love, K. T., Mahon, K. P., Levins, C. G., Whitehead, K. A., Querbes, W., Dorkin, J. R., Qin, J., Cantley, W., Qin, L. L., Racie, T., et al. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0910603106 · Cited by 969 · see citing works

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T., & Anderson, D. G. (2014) · Nature Biotechnology · doi:10.1038/nbt.2884 · Cited by 951 · see citing works

Multiplex genome editing to generate universal CAR T cells resistant to PD1 inhibition

Ren, J., Liu, X., Fang, C., Jiang, S., June, C. H., & Zhao, Y. (2017) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-16-1300 · Cited by 942 · see citing works

Actively personalized vaccination trial for newly diagnosed glioblastoma

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CAR T Cells Produced in vivo to Treat Cardiac Injury

Rurik, J. G., Tombácz, I., Yadegari, A., Fernández, P. O. M., Shewale, S. V., Li, L., Kimura, T., Soliman, O. Y., Papp, T. E., Tam, Y. K., et al. (2023) · HAL · Cited by 926 · see citing works

The functions and regulation of the PTEN tumour suppressor: new modes and prospects

Lee, Y.-R., Chen, M., & Pandolfi, P. P. (2018) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-018-0015-0 · Cited by 917 · see citing works

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

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Tumor Suppression in the Absence of p53-Mediated Cell-Cycle Arrest, Apoptosis, and Senescence

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Chemistry of Lipid Nanoparticles for RNA Delivery

Eygeris, Y., Gupta, M., Kim, J., & Sahay, G. (2021) · Accounts of Chemical Research · doi:10.1021/acs.accounts.1c00544 · Cited by 903 · see citing works

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

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Engineering circular RNA for potent and stable translation in eukaryotic cells

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The clinical progress of mRNA vaccines and immunotherapies

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Effects of single-dose interleukin-12 exposure on interleukin-12-associated toxicity and interferon-gamma production.

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Exploiting the Mutanome for Tumor Vaccination

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

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Interleukins in cancer: from biology to therapy

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Lipid nanoparticles enhance the efficacy of mRNA and protein subunit vaccines by inducing robust T follicular helper cell and humoral responses

Alameh, M.-G., Tombácz, I., Bettini, E., Lederer, K., Ndeupen, S., Sittplangkoon, C., Wilmore, J. R., Gaudette, B. T., Soliman, O. Y., Pine, M., et al. (2021) · Immunity · doi:10.1016/j.immuni.2021.11.001 · Cited by 775 · see citing works

Circular RNAs are long-lived and display only minimal early alterations in response to a growth factor

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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

Delivery of mRNA vaccines with heterocyclic lipids increases anti-tumor efficacy by STING-mediated immune cell activation

Miao, L., Li, L., Huang, Y., Delcassian, D., Chahal, J., Han, J., Shi, Y., Sadtler, K., Gao, W., Lin, J., et al. (2019) · Nature Biotechnology · doi:10.1038/s41587-019-0247-3 · Cited by 723 · see citing works

Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR–Cas gene editing

Liu, S., Cheng, Q., Wei, T., Yu, X., Johnson, L. T., Farbiak, L., & Siegwart, D. J. (2021) · Nature Materials · doi:10.1038/s41563-020-00886-0 · Cited by 653 · see citing works

COVID-19 vaccine development and a potential nanomaterial path forward

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Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T Cells

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Lipid-based Nanoparticles for Nucleic Acid Delivery

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Lipids and Lipid Derivatives for RNA Delivery

Zhang, Y., Sun, C., Wang, C., Jankovic, K. E., & Dong, Y. (2021) · Chemical Reviews · doi:10.1021/acs.chemrev.1c00244 · Cited by 638 · see citing works

Immune responses to gene therapy vectors: influence on vector function and effector mechanisms

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Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering

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Circular RNA Vaccines against SARS-CoV-2 and Emerging Variants

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CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy

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Unlocking the promise of mRNA therapeutics

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Chromatin Remodeling at DNA Double-Strand Breaks

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T Cells Expressing Chimeric Antigen Receptors Can Cause Anaphylaxis in Humans

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

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The tangled history of mRNA vaccines

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Identification of neoantigens for individualized therapeutic cancer vaccines

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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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Clinical advances and ongoing trials of mRNA vaccines for cancer treatment

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Tetanus toxoid and CCL3 improve dendritic cell vaccines in mice and glioblastoma patients

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Nanotechnology approaches for global infectious diseases

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Activity of Mesothelin-Specific Chimeric Antigen Receptor T Cells Against Pancreatic Carcinoma Metastases in a Phase 1 Trial

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Self-assembled mRNA vaccines

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A comparison of non-integrating reprogramming methods

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The landscape of mRNA nanomedicine

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Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs

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Self-amplifying RNA vaccines give equivalent protection against influenza to mRNA vaccines but at much lower doses

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Self-amplifying RNA vaccines for infectious diseases

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Engineering circular RNA for enhanced protein production

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