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 in vitro synthesis of biologically active RNA and RNA hybridization probes from plasmids containing a bacteriophage SP6 promoter

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Neoantigens in cancer immunotherapy

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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

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

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

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An immunogenic personal neoantigen vaccine for patients with melanoma

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

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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

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

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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

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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

Progress and challenges towards targeted delivery of cancer therapeutics

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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

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

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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

Loss of PTEN Promotes Resistance to T Cell–Mediated Immunotherapy

Peng, W., Chen, J. Q., Liu, C., Malu, S., Creasy, C., Tetzlaff, M. T., Xu, C., McKenzie, J. A., Zhang, C., Liang, X., et al. (2016) · Cancer Discovery · doi:10.1158/2159-8290.cd-15-0283 · Cited by 1,539

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

CRISPR-engineered T cells in patients with refractory cancer

Stadtmauer, E. A., Fraietta, J. A., Davis, M. M., Cohen, A. D., Weber, K. L., Lancaster, E., Mangan, P. A., Kulikovskaya, I., Gupta, M., Chen, F., et al. (2020) · Science · doi:10.1126/science.aba7365 · Cited by 1,478

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

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

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

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

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 1,218

Polymers for the sustained release of proteins and other macromolecules

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

Personalized vaccines for cancer immunotherapy

Şahin, U., & Türeci, Ö. (2018) · Science · doi:10.1126/science.aar7112 · Cited by 1,175

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

Akinc, A., Zumbuehl, A., Goldberg, M., Leshchiner, E. S., Busini, V., Hossain, N., Bacallado, S. A., Nguyen, D. N., Fuller, J., Alvarez, R., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1402 · Cited by 1,150

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 1,145

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

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

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

Principles of Tumor Suppression

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

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

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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

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

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

Actively personalized vaccination trial for newly diagnosed glioblastoma

Hilf, N., Kuttruff-Coqui, S., Frenzel, K., Bukur, V., Stevanović, S., Gouttefangeas, C., Platten, M., Tabatabai, G., Dutoit, V., van der Burg, S. H., et al. (2019) · Nature · doi:10.1038/s41586-018-0810-y · Cited by 938

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 937

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

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 917

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

Li, T., Kon, N., Jiang, L., Tan, M., Ludwig, T., Zhao, Y., Baer, R., & Gu, W. (2012) · Cell · doi:10.1016/j.cell.2012.04.026 · Cited by 912

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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Interleukins in cancer: from biology to therapy

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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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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

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

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

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Lipid-based Nanoparticles for Nucleic Acid Delivery

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COVID-19 vaccine development and a potential nanomaterial path forward

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

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

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

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Lipids and Lipid Derivatives for RNA Delivery

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

Billingsley, M. M., Singh, N., Ravikumar, P., Zhang, R., June, C. H., & Mitchell, M. J. (2020) · Nano Letters · doi:10.1021/acs.nanolett.9b04246 · Cited by 636

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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T Cells Expressing Chimeric Antigen Receptors Can Cause Anaphylaxis in Humans

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Chromatin Remodeling at DNA Double-Strand Breaks

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The tangled history of mRNA vaccines

Dolgin, E. (2021) · Nature · doi:10.1038/d41586-021-02483-w · Cited by 578

Identification of neoantigens for individualized therapeutic cancer vaccines

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

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Clinical advances and ongoing trials of mRNA vaccines for cancer treatment

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

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

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A comparison of non-integrating reprogramming methods

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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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The landscape of mRNA nanomedicine

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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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In vitro-transcribed antigen receptor mRNA nanocarriers for transient expression in circulating T cells in vivo

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CRISPR in cancer biology and therapy

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mRNA vaccine–induced neoantigen-specific T cell immunity in patients with gastrointestinal cancer

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Lipid nanoparticle-mediated lymph node–targeting delivery of mRNA cancer vaccine elicits robust CD8 + T cell response

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