Non-liver mRNA Delivery

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

Polack, F. P., Thomas, S. J., Kitchin, N., Absalon, J., Gurtman, A., Lockhart, S., Perez, J. L., Pérez Marc, G., Moreira, E. D., Zerbini, C., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2034577 · Cited by 14,592 · see citing works

Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine

Baden, L. R., El Sahly, H. M., Essink, B., Kotloff, K., Frey, S., Novak, R., Diemert, D., Spector, S. A., Rouphael, N., Creech, C. B., et al. (2021) · New England Journal of Medicine · doi:10.1056/nejmoa2035389 · Cited by 10,119 · see citing works

Direct Gene Transfer into Mouse Muscle in Vivo

Wolff, J. A., Malone, R. W., Williams, P., Chong, W., Acsadi, G., Jani, A., & Felgner, P. L. (1990) · Science · doi:10.1126/science.1690918 · Cited by 4,173 · see citing works

Understanding the nanoparticle–protein corona using methods to quantify exchange rates and affinities of proteins for nanoparticles

Cedervall, T., Lynch, I., Lindman, S., Berggård, T., Thulin, E., Nilsson, H., Dawson, K. A., & Linse, S. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0608582104 · Cited by 3,281 · see citing works

An mRNA Vaccine against SARS-CoV-2 — Preliminary Report

Jackson, L. A., Anderson, E. J., Rouphael, N. G., Roberts, P. C., Makhene, M., Coler, R. N., McCullough, M. P., Chappell, J. D., Denison, M. R., Stevens, L. J., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2022483 · Cited by 3,237 · see citing works

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

Adams, D., Gonzalez-Duarte, A., O’Riordan, W. D., Yang, C.-C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716153 · Cited by 3,047 · see citing works

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

Warren, L., Manos, P. D., Ahfeldt, T., Loh, Y.-H., Li, H., Lau, F., Ebina, W., Mandal, P. K., Smith, Z. D., Meissner, A., et al. (2010) · Cell Stem Cell · doi:10.1016/j.stem.2010.08.012 · Cited by 2,662 · 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

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

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

Karikó, K., Muramatsu, H., Welsh, F. A., Ludwig, J., Kato, H., Akira, S., & Weissman, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.200 · Cited by 1,930 · 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

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

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Gillmore, J. D., Gane, E., Taubel, J., Kao, J., Fontana, M., Maitland, M. L., Seitzer, J., O’Connell, D., Walsh, K. R., Wood, K., et al. (2021) · New England Journal of Medicine · doi:10.1056/nejmoa2107454 · Cited by 1,766 · 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

Distinct conformational states of SARS-CoV-2 spike protein

Cai, Y., Zhang, J., Xiao, T., Peng, H., Sterling, S. M., Walsh, R. M., Rawson, S., Rits-Volloch, S., & Chen, B. (2020) · Science · doi:10.1126/science.abd4251 · Cited by 1,221 · see citing works

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

mRNA is an endogenous ligand for toll-like receptor 3

Karikó, K., Ni, H., Capodici, J., Lamphier, M., & Weissman, D. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.m310175200 · Cited by 1,062 · see citing works

Mechanism of hard-nanomaterial clearance by the liver

Tsoi, K. M., MacParland, S. A., Ma, X., Spetzler, V. N., Echeverri, J. A., Ouyang, B., Fadel, S. M., Sykes, E. A., Goldaracena, N., Kaths, J. M., et al. (2016) · Nature Materials · doi:10.1038/nmat4718 · Cited by 1,020 · 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

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

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

Musunuru, K., Chadwick, A. C., Mizoguchi, T., Garcia, S. P., DeNizio, J. E., Reiss, C. W., Wang, K., Iyer, S., Dutta, C., Clendaniel, V., et al. (2021) · Nature · doi:10.1038/s41586-021-03534-y · Cited by 793 · see citing works

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

Principles for targeting RNA with drug-like small molecules

Warner, K. D., Hajdin, C. E., & Weeks, K. M. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.93 · Cited by 742 · see citing works

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

Kormann, M. S. D., Hasenpusch, G., Aneja, M. K., Nica, G., Flemmer, A. W., Herber-Jonat, S., Huppmann, M., Mays, L. E., Illenyi, M., Schams, A., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1733 · Cited by 733 · see citing works

Lipoproteins and their receptors in the central nervous system. Characterization of the lipoproteins in cerebrospinal fluid and identification of apolipoprotein B,E(LDL) receptors in the brain.

Pitas, R. E., Boyles, J. K., Lee, S. H., Hui, D., & Weisgraber, K. H. (1987) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(18)47945-8 · Cited by 714 · 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

Messenger RNA modifications: Form, distribution, and function

Gilbert, W. V., Bell, T. A., & Schaening-Burgos, C. (2016) · Science · doi:10.1126/science.aad8711 · Cited by 630 · see citing works

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

Wei, T., Cheng, Q., Min, Y.-L., Olson, E. N., & Siegwart, D. J. (2020) · Nature Communications · doi:10.1038/s41467-020-17029-3 · Cited by 603 · see citing works

Elimination Pathways of Nanoparticles

Poon, W., Zhang, Y.-N., Ouyang, B., Kingston, B. R., Wu, J. L. Y., Wilhelm, S., & Chan, W. C. W. (2019) · ACS Nano · doi:10.1021/acsnano.9b01383 · Cited by 578 · see citing works

Recognition of Endogenous Nucleic Acids by the Innate Immune System

Roers, A., Hiller, B., & Hornung, V. (2016) · Immunity · doi:10.1016/j.immuni.2016.04.002 · Cited by 541 · see citing works

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

A noninflammatory mRNA vaccine for treatment of experimental autoimmune encephalomyelitis

Krienke, C., Kolb, L., Diken, E., Streuber, M., Kirchhoff, S., Bukur, T., Akilli-Öztürk, Ö., Kranz, L. M., Berger, H., Petschenka, J., et al. (2021) · Science · doi:10.1126/science.aay3638 · Cited by 520 · see citing works

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

Lokugamage, M. P., Vanover, D. A., Beyersdorf, J. P., Hatit, M. Z. C., Rotolo, L., Echeverri, E. S., Peck, H. E., Ni, H., Yoon, J., Kim, Y., et al. (2021) · Nature Biomedical Engineering · doi:10.1038/s41551-021-00786-x · Cited by 507 · see citing works

The biomolecular corona is retained during nanoparticle uptake and protects the cells from the damage induced by cationic nanoparticles until degraded in the lysosomes

Wang, F., Yu, L., Monopoli, M. P., Sandin, P., Mahon, E., Salvati, A. L., & Dawson, K. A. (2013) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2013.04.010 · Cited by 426 · see citing works

Mammalian Retrovirus-like Protein PEG10 Packages Its Own MRNA and Can Be Pseudotyped for MRNA Delivery

Segel, M., Lash, B., Song, J., Ladha, A., Liu, C. C., Jin, X., Mekhedov, S. L., Macrae, R. K., Koonin, E. V., & Zhang, F. (2021) · Science · doi:10.1126/science.abg6155 · Cited by 423 · see citing works

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

Qiu, M., Glass, Z., Chen, J., Haas, M., Jin, X., Zhao, X., Rui, X., Ye, Z., Li, Y., Zhang, F., et al. (2021) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2020401118 · Cited by 419 · 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

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

Rothgangl, T., Dennis, M. K., Lin, P. J. C., Oka, R., Witzigmann, D., Villiger, L., Qi, W., Hruzova, M., Kissling, L., Lenggenhager, D., et al. (2021) · Nature Biotechnology · doi:10.1038/s41587-021-00933-4 · Cited by 378 · see citing works

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

Liu, J., Chang, J., Jiang, Y., Meng, X., Sun, T., Mao, L., Xu, Q., & Wang, M. (2019) · Advanced Materials · doi:10.1002/adma.201902575 · Cited by 373 · see citing works

Improving mRNA-Based Therapeutic Gene Delivery by Expression-Augmenting 3′ UTRs Identified by Cellular Library Screening

Orlandini von Niessen, A. G., Poleganov, M. A., Rechner, C., Plaschke, A., Kranz, L. M., Fesser, S., Diken, M., Löwer, M., Vallazza, B., Beissert, T., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2018.12.011 · Cited by 365 · see citing works

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

Dahlman, J. E., Kauffman, K. J., Xing, Y., Shaw, T. E., Mir, F. F., Dlott, C. C., Langer, R., Anderson, D. G., & Wang, E. T. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1620874114 · Cited by 335 · see citing works

Lipid Nanoparticle Formulations for Enhanced Co-delivery of siRNA and mRNA

Ball, R. L., Hajj, K. A., Vizelman, J., Bajaj, P., & Whitehead, K. A. (2018) · Nano Letters · doi:10.1021/acs.nanolett.8b01101 · Cited by 314 · see citing works

High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

Sago, C. D., Lokugamage, M. P., Paunovska, K., Vanover, D. A., Monaco, C. M., Shah, N. N., Gamboa Castro, M., Anderson, S. E., Rudoltz, T. G., Lando, G. N., et al. (2018) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1811276115 · Cited by 313 · see citing works

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

Veiga, N., Goldsmith, M., Granot, Y., Rosenblum, D., Dammes, N., Kedmi, R., Ramishetti, S., & Peer, D. (2018) · Nature Communications · doi:10.1038/s41467-018-06936-1 · Cited by 308 · 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

A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation

Paunovska, K., Sago, C. D., Monaco, C. M., Hudson, W. H., Castro, M. G., Rudoltz, T. G., Kalathoor, S., Vanover, D. A., Santangelo, P. J., Ahmed, R., et al. (2018) · Nano Letters · doi:10.1021/acs.nanolett.8b00432 · Cited by 277 · see citing works

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

Robinson, E., MacDonald, K. D., Slaughter, K., McKinney, M., Patel, S., Sun, C., & Sahay, G. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.05.014 · Cited by 272 · see citing works

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

Vaidyanathan, S., Azizian, K. T., Haque, A. K. M. A., Henderson, J. M., Hendel, A., Shore, S., Antony, J. S., Hogrefe, R. I., Kormann, M. S. D., Porteus, M. H., et al. (2018) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2018.06.010 · Cited by 262 · see citing works

Lipid nanoparticles for delivery of messenger RNA to the back of the eye

Patel, S., Ryals, R. C., Weller, K. K., Pennesi, M. E., & Sahay, G. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.04.015 · Cited by 260 · 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

PECAM-1 directed re-targeting of exogenous mRNA providing two orders of magnitude enhancement of vascular delivery and expression in lungs independent of apolipoprotein E-mediated uptake

Parhiz, H., Shuvaev, V. V., Pardi, N., Khoshnejad, M., Kiseleva, R. Y., Brenner, J. S., Uhler, T., Tuyishime, S., Mui, B. L., Tam, Y. K., et al. (2018) · Journal of Controlled Release · doi:10.1016/j.jconrel.2018.10.015 · Cited by 233 · see citing works

Visualization of early events in mRNA vaccine delivery in non-human primates via PET–CT and near-infrared imaging

Lindsay, K. E., Bhosle, S. M., Zurla, C., Beyersdorf, J., Rogers, K. A., Vanover, D., Xiao, P., Araínga, M., Shirreff, L. M., Pitard, B., et al. (2019) · Nature Biomedical Engineering · doi:10.1038/s41551-019-0378-3 · Cited by 218 · see citing works

Treatment of influenza and SARS-CoV-2 infections via mRNA-encoded Cas13a in rodents

Blanchard, E. L., Vanover, D., Bawage, S., Tiwari, P., Rotolo, L., Beyersdorf, J. P., Peck, H., Bruno, N. C., Hincapie, R., Michel, F., et al. (2021) · Nature Biotechnology · doi:10.1038/s41587-021-00822-w · Cited by 212 · see citing works

Nanoparticles Containing Oxidized Cholesterol Deliver mRNA to the Liver Microenvironment at Clinically Relevant Doses

Paunovska, K., Da Silva Sanchez, A. J., Sago, C. D., Gan, Z., Lokugamage, M. P., Islam, F. Z., Kalathoor, S., Krupczak, B. R., & Dahlman, J. E. (2019) · Advanced Materials · doi:10.1002/adma.201807748 · Cited by 205 · see citing works

The effects of PEGylation on LNP based mRNA delivery to the eye

Ryals, R. C., Patel, S., Acosta, C., McKinney, M., Pennesi, M. E., & Sahay, G. (2020) · PLOS ONE · doi:10.1371/journal.pone.0241006 · Cited by 194 · see citing works

Selective targeting of nanomedicine to inflamed cerebral vasculature to enhance the blood–brain barrier

Marcos-Contreras, O. A., Greineder, C. F., Kiseleva, R. Y., Parhiz, H., Walsh, L. R., Zuluaga‐Ramirez, V., Myerson, J. W., Hood, E. D., Villa, C. H., Tombácz, I., et al. (2020) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1912012117 · Cited by 187 · see citing works

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

Zhang, X., Zhao, W., Nguyen, G. N., Zhang, C., Zeng, C., Yan, J., Du, S., Hou, X., Li, W., Jiang, J., et al. (2020) · Science Advances · doi:10.1126/sciadv.abc2315 · Cited by 183 · see citing works

mRNA transfection of dendritic cells: Synergistic effect of ARCA mRNA capping with Poly(A) chains in cis and in trans for a high protein expression level

Mockey, M., Gonçalves, C., Dupuy, F. P., Lemoine, F. M., Pichon, C., & Midoux, P. (2005) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2005.12.105 · Cited by 165 · see citing works

MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

Jain, R., Frederick, J. P., Huang, E. Y., Burke, K. E., Mauger, D. M., Andrianova, E. A., Farlow, S. J., Siddiqui, S., Pimentel, J., Cheung-Ong, K., et al. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0734 · Cited by 164 · see citing works

Analyzing 2000 <i>in Vivo</i> Drug Delivery Data Points Reveals Cholesterol Structure Impacts Nanoparticle Delivery

Paunovska, K., Gil, C. J., Lokugamage, M. P., Sago, C. D., Sato, M., Lando, G. N., Gamboa Castro, M., Bryksin, A. V., & Dahlman, J. E. (2018) · ACS Nano · doi:10.1021/acsnano.8b03640 · Cited by 160 · see citing works

Constrained Nanoparticles Deliver siRNA and sgRNA to T Cells In Vivo without Targeting Ligands

Lokugamage, M. P., Sago, C. D., Gan, Z., Krupczak, B. R., & Dahlman, J. E. (2019) · Advanced Materials · doi:10.1002/adma.201902251 · Cited by 155 · see citing works

Leveraging mRNA Sequences and Nanoparticles to Deliver SARS‐CoV‐2 Antigens In Vivo

Zeng, C., Hou, X., Yan, J., Zhang, C., Li, W., Zhao, W., Du, S., & Dong, Y. (2020) · Advanced Materials · doi:10.1002/adma.202004452 · Cited by 152 · see citing works

Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles

Hatit, M. Z. C., Lokugamage, M. P., Dobrowolski, C. N., Paunovska, K., Ni, H., Zhao, K., Vanover, D. A., Beyersdorf, J. P., Peck, H. E., Loughrey, D., et al. (2022) · Nature Nanotechnology · doi:10.1038/s41565-021-01030-y · Cited by 149 · see citing works

Theranostic barcoded nanoparticles for personalized cancer medicine

Yaari, Z. A., da Silva, D., Zinger, A., Goldman, E., Kajal, A., Tshuva, R., Barak, E., Dahan, N., Hershkovitz, D., Goldfeder, M., et al. (2016) · Nature Communications · doi:10.1038/ncomms13325 · Cited by 147 · see citing works

Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

Guan, S., Munder, A., Hedtfeld, S., Braubach, P., Glage, S., Zhang, L., Lienenklaus, S., Schultze, A., Hasenpusch, G., Garrels, W., et al. (2019) · Nature Nanotechnology · doi:10.1038/s41565-018-0358-x · Cited by 130 · see citing works

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

Sago, C. D., Lokugamage, M. P., Islam, F. Z., Krupczak, B. R., Sato, M., & Dahlman, J. E. (2018) · Journal of the American Chemical Society · doi:10.1021/jacs.8b08976 · Cited by 129 · see citing works

Mild Innate Immune Activation Overrides Efficient Nanoparticle‐Mediated RNA Delivery

Lokugamage, M. P., Gan, Z., Zurla, C., Levin, J., Islam, F. Z., Kalathoor, S., Sato, M., Sago, C. D., Santangelo, P. J., & Dahlman, J. E. (2020) · Advanced Materials · doi:10.1002/adma.201904905 · Cited by 126 · see citing works

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

Zhang, X., Li, B., Luo, X., Zhao, W., Jiang, J., Zhang, C., Gao, M., Chen, X., & Dong, Y. (2017) · ACS Applied Materials &amp; Interfaces · doi:10.1021/acsami.7b08163 · Cited by 107 · see citing works

Rapid, Single-Cell Analysis and Discovery of Vectored mRNA Transfection In Vivo with a loxP-Flanked tdTomato Reporter Mouse

Kauffman, K. J., Oberli, M. A., Dorkin, J. R., Hurtado, J. E., Kaczmarek, J. C., Bhadani, S., Wyckoff, J., Langer, R., Jaklenec, A., & Anderson, D. G. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.11.005 · Cited by 103 · see citing works

Engineering Caveolae-Targeted Lipid Nanoparticles To Deliver mRNA to the Lungs

Li, Q., Chan, C. S., Peterson, N. C., Hanna, R. N., Alfaro, A., Allen, K. L., Wu, H., Dall’Acqua, W. F., Borrok, M. J., & Santos, J. L. (2020) · ACS Chemical Biology · doi:10.1021/acschembio.0c00003 · Cited by 103 · see citing works

A Nanoprimer To Improve the Systemic Delivery of siRNA and mRNA

Saunders, N., Paolini, M. S., Fenton, O. S., Poul, L., Devallière, J., Mpambani, F., Darmon, A., Bergère, M., Jibault, O., Germain, M., et al. (2020) · Nano Letters · doi:10.1021/acs.nanolett.0c00752 · Cited by 97 · see citing works

5′-Phosphorothiolate Dinucleotide Cap Analogues: Reagents for Messenger RNA Modification and Potent Small-Molecular Inhibitors of Decapping Enzymes

Wojtczak, B. A., Sikorski, P. J., Fac-Dabrowska, K., Nowicka, A. M., Warmiński, M., Kubacka, D., Nowak, E., Nowotny, M., Kowalska, J., & Jemielity, J. (2018) · Journal of the American Chemical Society · doi:10.1021/jacs.8b02597 · Cited by 95 · see citing works

Locked Nucleic Acid (LNA)-Modified Dinucleotide mRNA Cap Analogue: Synthesis, Enzymatic Incorporation, and Utilization

Kore, A. R., Shanmugasundaram, M., Charles, I., Vlassov, A. V., & Barta, T. J. (2009) · Journal of the American Chemical Society · doi:10.1021/ja901655p · Cited by 90 · see citing works

mRNA secondary structure optimization using a correlated stem–loop prediction

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In Vivo Introduction of mRNA Encapsulated in Lipid Nanoparticles to Brain Neuronal Cells and Astrocytes via Intracerebroventricular Administration

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Lipid nanoparticles deliver mRNA to the blood–brain barrier

Kuzminich, Y., Shakked, A., Calkins, R., Rudden, S., Jones, C., Doan, J., Jang, B., Echeverri, E. S., Zenhausern, R., Lian, L., et al. (2024) · Nano Research · doi:10.1007/s12274-024-6827-7 · Cited by 23 · see citing works

Advancing mRNA Therapeutics: The Role and Future of Nanoparticle Delivery Systems

Li, J., Zhang, Y., Yang, Y., & Sun, T. (2024) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.4c00276 · Cited by 23 · see citing works

Interaction design in mRNA delivery systems

Yu, M., Lin, L., Zhou, D., & Liu, S. (2024) · Journal of Controlled Release · doi:10.1016/j.jconrel.2024.11.038 · Cited by 23 · see citing works

Non‐Viral Gene Delivery Systems: Current Advances and Therapeutic Applications

Wang, F., Liang, L., Wang, C., Zhang, C., Ye, Y., Dou, L., Shi, L., Zhou, D., & Sun, X. (2025) · Chemistry - An Asian Journal · doi:10.1002/asia.202500684 · Cited by 21 · see citing works

Optimization of a Liposomal DNase I Formulation with an Extended Circulating Half-Life

Arias, L. C. P., Huynh, D. N., Babity, S., Marleau, S., & Brambilla, D. (2022) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.2c00086 · Cited by 21 · see citing works

The Development of Cell-Penetrating Peptides for Efficient and Selective In Vivo Expression of mRNA in Spleen Tissue

Porosk, L., Härk, H. H., Arukuusk, P., Haljasorg, U., Peterson, P., & Kurrikoff, K. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15030952 · Cited by 21 · see citing works

The clinical progress and challenges of mRNA vaccines

Yu, M., Wang, N., Zhu, J., & Lin, Y. (2023) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1894 · Cited by 22 · see citing works

Aerosol Inhalation of Gene Delivery Therapy for Pulmonary Diseases

Huang, Y., Zhang, J., Wang, X., Jing, H., & Li, H. (2024) · Biomolecules · doi:10.3390/biom14080904 · Cited by 20 · see citing works

Lipid nanoparticle-mediated targeted mRNA delivery and its application in cancer therapy

Sui, Y., Hou, X., Zhang, J., Hong, X., Wang, H., Xiao, Y., & Zeng, X. (2025) · Journal of Materials Chemistry B · doi:10.1039/d5tb01556a · Cited by 23 · see citing works

Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides

Wickline, S. A., Hou, K. K., & Pan, H. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24119455 · Cited by 20 · see citing works

Differences and Similarities of the Intravenously Administered Lipid Nanoparticles in Three Clinical Trials: Potential Linkage between Lipid Nanoparticles and Extracellular Vesicles

Suzuki, Y., Katsurada, Y., & Hyodo, K. (2023) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.3c00547 · Cited by 21 · see citing works

A review of RNA nanoparticles for drug/gene/protein delivery in advanced therapies: Current state and future prospects

Vosoughi, P., Naghib, S. M., Kangarshahi, B. M., & Mozafari, M. R. (2025) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2025.139532 · Cited by 20 · see citing works

In Silico Screening Accelerates Nanocarrier Design for Efficient mRNA Delivery

Henser‐Brownhill, T., Martin, L., Samangouei, P., Ladak, A., Apostolidou, M., Nagel, B., & Kwok, A. (2024) · Advanced Science · doi:10.1002/advs.202401935 · Cited by 18 · see citing works

Nanoparticle delivery of a prodrug-activating bacterial enzyme leads to anti-tumor responses

Huayamares, S. G., Lian, L., Rab, R., Hou, Y., Radmand, A., Kim, H., Zenhausern, R., Achyut, B. R., Gilbert‐Ross, M., Lokugamage, M. P., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-58548-1 · Cited by 18 · see citing works

Emerging mRNA Technology for Liver Disease Therapy

Wang, J., Fang, Y., Luo, Z., Wang, J., & Zhao, Y. (2024) · ACS Nano · doi:10.1021/acsnano.4c02987 · Cited by 19 · see citing works

A Single‐Dose mRNA Vaccine Employing Porous Silica Nanoparticles Induces Robust Immune Responses Against the Zika Virus

Shin, H., Kang, S., Won, C., & Min, D. (2024) · Advanced Science · doi:10.1002/advs.202404590 · Cited by 18 · see citing works

Nucleic acid delivery by lipid nanoparticles for organ targeting

Guo, J., Gu, M., Chen, Y., Xiong, T., Zhang, Y., Chen, S., Li, M., Chen, X., & Peng, X. (2025) · Chinese Chemical Letters · doi:10.1016/j.cclet.2025.110849 · Cited by 18 · see citing works

Critical considerations of mRNA–LNP technology for CAR-T therapy: components, payloads and emerging horizons

Qu, Y., Liu, R., Sun, D., & Dai, Z. (2024) · Materials Chemistry Frontiers · doi:10.1039/d4qm00479e · Cited by 17 · see citing works

Delivery of mRNA for cancer therapy: progress and prospects

Su, L.-J., Xu, M.-X., Ji, Z.-H., Zhu, J.-Q., Yu, M., Wang, Y., & Lin, Y. (2023) · Nano Today · doi:10.1016/j.nantod.2023.102013 · Cited by 17 · see citing works

Exciting Times for Lipid Nanoparticles: How Canadian Discoveries Are Enabling Gene Therapies

Cheng, M. H. Y., Brimacombe, C. A., Verbeke, R., & Cullis, P. R. (2022) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.2c00365 · Cited by 17 · see citing works

Cell-Derived Vesicles for mRNA Delivery

Li, Z., Liu, Z., Wu, J., & Li, B. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14122699 · Cited by 16 · see citing works

Quaternization drives spleen-to-lung tropism conversion for mRNA-loaded lipid-like nanoassemblies

Huang, Y., Wu, J., Li, S., Liu, Z., Li, Z., Zhou, B., & Li, B. (2024) · Theranostics · doi:10.7150/thno.90071 · Cited by 16 · see citing works

One‐Component Cationic Lipids for Systemic mRNA Delivery to Splenic T Cells

Zhang, X., Su, K., Wu, S., Lin, L., He, S., Yan, X., Shi, L., & Liu, S. (2024) · Angewandte Chemie · doi:10.1002/ange.202405444 · Cited by 16 · see citing works

Chemical Design Strategy of Ionizable Lipids for In Vivo mRNA Delivery

He, Z., Liu, Z., & Chen, Y. (2024) · ChemMedChem · doi:10.1002/cmdc.202400199 · Cited by 16 · see citing works

Branching in poly(amine-co-ester) polyplexes impacts mRNA transfection

Shin, K., Suh, H., Suberi, A., Whang, C., Ene, M., Grundler, J., Grun, M. K., & Saltzman, W. M. (2024) · Biomaterials · doi:10.1016/j.biomaterials.2024.122692 · Cited by 16 · see citing works

Nonsense Mutations in Rare and Ultra‐Rare Human Disorders: An Overview

Vitale, E., Ricci, D., Corrao, F., Fiduccia, I., Cruciata, I., Carollo, P. S., Branchini, A., Lentini, L., & Pibiri, I. (2025) · IUBMB Life · doi:10.1002/iub.70031 · Cited by 15 · see citing works

Glycolipid nanoparticles target the spleen and detarget the liver without charge

Gentry, K., Lian, L., Kim, H., Celik, O., Jones, C., Podilapu, A. R., Shakked, A., Loughrey, D., Zenhausern, R., Jang, B., et al. (2025) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2409569122 · Cited by 15 · see citing works

New trends in brain tumor immunity with the opportunities of lymph nodes targeted drug delivery

Qi, Y., Xiong, W., Chen, Q., Ye, Z., Jiang, C., He, Y., & Ye, Q. (2023) · Journal of Nanobiotechnology · doi:10.1186/s12951-023-02011-0 · Cited by 15 · see citing works

Nanocarriers for inhaled delivery of RNA therapeutics

Huayamares, S. G., Zenhausern, R., & Loughrey, D. (2024) · Current Research in Biotechnology · doi:10.1016/j.crbiot.2024.100200 · Cited by 17 · see citing works

Golden Threads of Lipid Nanoparticles (LNPs) contributing to COVID-19 mRNA Vaccine: A Review

Vasu, M., Yadav, P., Satyanarayana, Y., Ahlawat, J., Dulla, S., & Kumar, K. N. (2024) · BioNanoScience · doi:10.1007/s12668-024-01589-5 · Cited by 15 · see citing works

Polymers for mRNA Delivery

Wang, H., & Cheng, Y. (2025) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.70002 · Cited by 15 · see citing works

Therapeutic mRNA delivery with targeted lipid nanoparticles: next-generation transformative medicines

Iqbal, U. H., & Sandhu, J. K. (2022) · Cell and Gene Therapy Insights · doi:10.18609/cgti.2022.278

Nonviral targeted mRNA delivery: principles, progresses, and challenges

He, X., Li, G., Huang, L., Shi, H., Zhong, S., Zhao, S., Jiao, X., Xin, J., Yin, X., Liu, S., et al. (2025) · MedComm · doi:10.1002/mco2.70035 · Cited by 20 · see citing works

mRNA therapeutics: beyond vaccine applications

Bhat, B., Karve, S., & Anderson, D. G. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2021.05.004 · Cited by 28 · see citing works

mRNA-based vaccines and therapeutics: an in-depth survey of current and upcoming clinical applications

Wang, Y., Kumari, M., Chen, G., Hong, M.-H., Yuan, J. P., Tsai, J.-L., & Wu, H. (2023) · Journal of Biomedical Science · doi:10.1186/s12929-023-00977-5 · Cited by 183 · see citing works

Research progress on lipid nanoparticle messenger RNA delivery system

He, S., & Liu, S. (2025) · Journal of Zhejiang University (Medical Sciences) · doi:10.3724/zdxbyxb-2024-0709 · Cited by 1 · see citing works