Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Genome engineering using the CRISPR-Cas9 system

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Principles of nanoparticle design for overcoming biological barriers to drug delivery

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Analysis of nanoparticle delivery to tumours

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mRNA vaccines — a new era in vaccinology

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The Effect of Nanoparticle Size, Shape, and Surface Chemistry on Biological Systems

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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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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 · see citing works

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

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Toxicity of cationic lipids and cationic polymers in gene delivery

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RNA targeting with CRISPR–Cas13

Abudayyeh, O. O., Gootenberg, J. S., Essletzbichler, P., Han, S., Joung, J., Belanto, J. J., Verdine, V., Cox, D. B. T., Kellner, M. J., Regev, A., et al. (2017) · Nature · doi:10.1038/nature24049 · Cited by 2,093 · see citing works

Understanding the diversity of membrane lipid composition

原山, 武., & Riezman, H. (2018) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2017.138 · Cited by 2,066 · see citing works

Transferrin-functionalized nanoparticles lose their targeting capabilities when a biomolecule corona adsorbs on the surface

Salvati, A., Pitek, A. S., Monopoli, M. P., Prapainop, K., Bombelli, F. B., Hristov, D. R., Kelly, P. M., Åberg, C., Mahon, E., & Dawson, K. A. (2013) · Nature Nanotechnology · doi:10.1038/nnano.2012.237 · Cited by 1,912 · see citing works

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

Kosicki, M., Tomberg, K., & Bradley, A. (2018) · Nature Biotechnology · doi:10.1038/nbt.4192 · Cited by 1,883 · see citing works

Rational design of cationic lipids for siRNA delivery

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RNA editing with CRISPR-Cas13

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Combination of prostate-specific antigen, clinical stage, and Gleason score to predict pathological stage of localized prostate cancer. A multi-institutional update

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

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Overcoming the challenges in administering biopharmaceuticals: Formulation and delivery strategies

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Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

Gilleron, J., Querbes, W., Zeigerer, A., Borodovsky, A., Marsico, G., Schubert, U., Manygoats, K., Seifert, S., Andree, C., Stöter, M., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2612 · Cited by 1,681 · see citing works

Particle size-dependent organ distribution of gold nanoparticles after intravenous administration

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CRISPR-Cas guides the future of genetic engineering

Knott, G. J., & Doudna, J. A. (2018) · Science · doi:10.1126/science.aat5011 · Cited by 1,410 · see citing works

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 · see citing works

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

Haapaniemi, E., Botla, S., Persson, J., Schmierer, B., & Taipale, J. (2018) · Nature Medicine · doi:10.1038/s41591-018-0049-z · Cited by 1,264 · see citing works

Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms

Akinc, A., Querbes, W., De, S., Qin, J., Frank-Kamenetsky, M., Jayaprakash, K. N., Jayaraman, M., Rajeev, K. G., Cantley, W. L., Dorkin, J. R., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.85 · Cited by 1,261 · see citing works

CRISPR RNA–guided activation of endogenous human genes

Maeder, M. L., Linder, S. J., Cascio, V. M., Fu, Y., Ho, Q. H., & Joung, J. K. (2013) · Nature Methods · doi:10.1038/nmeth.2598 · Cited by 1,249 · 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

The Many Pathways of RNA Degradation

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Cell-Penetrating Peptides: From Basic Research to Clinics

Guidotti, G., Brambilla, L., & Rossi, D. (2017) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2017.01.003 · Cited by 1,158 · see citing works

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

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Codon Optimality Is a Major Determinant of mRNA Stability

Presnyak, V., Alhusaini, N., Chen, Y.-H., Martin, S., Morris, N., Kline, N., Olson, S., Weinberg, D., Baker, K. E., Graveley, B. R., et al. (2015) · Cell · doi:10.1016/j.cell.2015.02.029 · Cited by 1,078 · see citing works

DNA vaccines: ready for prime time?

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Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos

Niu, Y., Shen, B., Cui, Y., Chen, Y., Wang, J., Wang, L., Kang, Y., Zhao, X., Si, W., Li, W., et al. (2014) · Cell · doi:10.1016/j.cell.2014.01.027 · Cited by 1,033 · see citing works

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

Pardi, N., Tuyishime, S., Muramatsu, H., Kariko, K., Mui, B. L., Tam, Y. K., Madden, T. D., Hope, M. J., & Weissman, D. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.08.007 · Cited by 1,014 · see citing works

CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes

Komor, A. C., Badran, A. H., & Liu, D. R. (2016) · Cell · doi:10.1016/j.cell.2016.10.044 · Cited by 1,011 · see citing works

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

Sahay, G., Querbes, W., Alabi, C., Eltoukhy, A., Sarkar, S., Zurenko, C., Karagiannis, E., Love, K., Chen, D., Zoncu, R., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2614 · Cited by 948 · see citing works

Degradable poly (β-amino esters): synthesis, characterization, and self-assembly with plasmid DNA

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Modified mRNA vaccines protect against zika virus infection

Richner, J. M., Himansu, S., Dowd, K. A., Butler, S. L., Salazar, V., Fox, J. M., Julander, J. G., Tang, W. W., Shresta, S., Pierson, T. C., et al. (2017) · Cell · doi:10.1016/j.cell.2017.02.017 · Cited by 909 · see citing works

A STING-activating nanovaccine for cancer immunotherapy

Luo, M., Wang, H., Wang, Z., Cai, H., Lu, Z., Li, Y., Du, M., Huang, G., Wang, C., Chen, X., et al. (2017) · Nature Nanotechnology · doi:10.1038/nnano.2017.52 · Cited by 904 · see citing works

Engineering circular RNA for potent and stable translation in eukaryotic cells

Wesselhoeft, R. A., Kowalski, P. S., & Anderson, D. G. (2018) · Nature Communications · doi:10.1038/s41467-018-05096-6 · Cited by 884 · see citing works

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

Pardi, N., Hogan, M. J., Pelc, R. S., Muramatsu, H., Andersen, H., DeMaso, C. R., Dowd, K. A., Sutherland, L. L., Scearce, R. M., Parks, R., et al. (2017) · Nature · doi:10.1038/nature21428 · Cited by 884 · see citing works

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

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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

Exploiting the Mutanome for Tumor Vaccination

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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

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Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

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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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Nonviral delivery of self-amplifying RNA vaccines

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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 · see citing works

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

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Stability of Endogenous and Added RNA in Blood Specimens, Serum, and Plasma

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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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Correction of a genetic disease in mouse via use of CRISPR-Cas9

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Delivery technologies for genome editing

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

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Influenza Virus: Immunity and Vaccination Strategies. Comparison of the Immune Response to Inactivated and Live, Attenuated Influenza 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

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

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

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

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Liposomes as vaccine delivery systems: a review of the recent advances

Schwendener, R. A. (2014) · Therapeutic Advances in Vaccines · doi:10.1177/2051013614541440 · Cited by 524 · see citing works

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

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

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Recent Advances in Targeting CD8 T-Cell Immunity for More Effective Cancer Immunotherapy

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PAMAM dendrimers as efficient drug and gene delivery nanosystems for cancer therapy

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

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Regulation of antiviral T cell responses by type I interferons

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ELECTROSTATIC INTERACTIONS IN MEMBRANES AND PROTEINS

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Overcoming the PEG-addiction: well-defined alternatives to PEG, from structure–property relationships to better defined therapeutics

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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

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Materials for non-viral intracellular delivery of messenger RNA therapeutics

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Mechanism of Polyplex- and Lipoplex-Mediated Delivery of Nucleic Acids: Real-Time Visualization of Transient Membrane Destabilization without Endosomal Lysis

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Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems

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Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium

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Parallel Synthesis and Biophysical Characterization of a Degradable Polymer Library for Gene Delivery

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Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

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Engineering of CRISPR-Cas12b for human genome editing

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

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Engineering the delivery system for CRISPR-based genome editing

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Intracellular delivery of nanomaterials: How to catch endosomal escape in the act

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Restoration of tumour-growth suppression in vivo via systemic nanoparticle-mediated delivery of PTEN mRNA

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

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

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Untitled work

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