Alvarez, E. M. et al. The global burden of adolescent and young adult cancer in 2019: a systematic analysis for the Global Burden of Disease Study 2019. Lancet Oncol. 23, 27–52 (2022).
Global Burden of Disease 2019 Cancer Collaboration. Cancer incidence, mortality, years of life lost, years lived with disability, and disability-adjusted life years for 29 cancer groups from 2010 to 2019: a systematic analysis for the Global Burden of Disease Study 2019. JAMA Oncol. https://doi.org/10.1001/jamaoncol.2021.6987 (2021).
Google Scholar
Vos, T. et al. Global burden of 369 diseases and injuries in 204 countries and territories, 1990–2019: a systematic analysis for the Global Burden of Disease Study 2019. Lancet 396, 1204–1222 (2020).
Roth, A. G. et al. Global burden of cardiovascular diseases and risk factors, 1990–2019. J. Am. Coll. Cardiol. 76, 2982–3021 (2020).
Forouzanfar, M. H. et al. Global, regional, and national comparative risk assessment of 79 behavioural, environmental and occupational, and metabolic risks or clusters of risks, 1990–2015: a systematic analysis for the Global Burden of Disease Study 2015. Lancet 388, 1659–1724 (2016).
Frank, T. D. et al. Global, regional, and national incidence, prevalence, and mortality of HIV, 1980–2017, and forecasts to 2030, for 195 countries and territories: a systematic analysis for the Global Burden of Diseases, Injuries, and Risk Factors Study 2017. Lancet HIV 6, e831–e859 (2019).
Watson, D. J. The human genome project: past, present, and future. Science 248, 44–49 (1990).
Google Scholar
Fire, A. et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391, 806–811 (1998).
Google Scholar
Martin, J. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816–821 (2012).
Google Scholar
Shahryari, A. et al. Development and clinical translation of approved gene therapy products for genetic disorders. Front. Genet. 10, 868 (2019).
Polack, F. P. et al. Safety and efficacy of the BNT162b2 mRNA COVID-19 vaccine. N. Engl. J. Med. 383, 2603–2615 (2020).
Gillmore, J. D. et al. CRISPR–Cas9 in vivo gene editing for transthyretin amyloidosis. N. Engl. J. Med. 385, 493–502 (2021).
Ruan, G.-X. et al. CRISPR/Cas9-mediated genome editing as a therapeutic approach for Leber congenital amaurosis 10. Mol. Ther. 25, 331–341 (2017).
McKay, P. F. et al. Self-amplifying RNA SARS-CoV-2 lipid nanoparticle vaccine candidate induces high neutralizing antibody titers in mice. Nat. Commun. 11, 3523 (2020).
Google Scholar
Wesselhoeft, R. A., Kowalski, P. S. & Anderson, D. G. Engineering circular RNA for potent and stable translation in eukaryotic cells. Nat. Commun. 9, 2629 (2018).
Google Scholar
Dangouloff, T. & Servais, L. Clinical evidence supporting early treatment of patients with spinal muscular atrophy: current perspectives. Ther. Clin. Risk Manag. 15, 1153–1161 (2019).
Kay, M. A. State-of-the-art gene-based therapies: the road ahead. Nat. Rev. Genet. 12, 316–328 (2011).
Urits, I. et al. A review of patisiran (Onpattro®) for the treatment of polyneuropathy in people with hereditary transthyretin amyloidosis. Neurol. Ther. 9, 301–315 (2020).
Dunbar, E. C. et al. Gene therapy comes of age. Science 359, eaan4672 (2018).
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M. & Sah, D. W. Y. Gene therapy for neurological disorders: progress and prospects. Nat. Rev. Drug Discov. 17, 641–659 (2018).
Jones, C. H., Chen, C.-K., Ravikrishnan, A., Rane, S. & Pfeifer, B. A. Overcoming nonviral gene delivery barriers: perspective and future. Mol. Pharm. 10, 4082–4098 (2013).
Malaviya, M. & Shiroya, M. Systemic gene delivery using lipid envelope systems and its potential in overcoming challenges. Int. J. Pharm. Drug Anal. 9, 46–55 (2021).
Uchida, S. & Kataoka, K. Design concepts of polyplex micelles for in vivo therapeutic delivery of plasmid DNA and messenger RNA. J. Biomed. Mater. Res. A 107, 978–990 (2019).
Sung, Y. K. & Kim, S. W. Recent advances in the development of gene delivery systems. Biomater. Res. 23, 8 (2019).
Patil, S. D., Rhodes, D. G. & Burgess, D. J. DNA-based therapeutics and DNA delivery systems: a comprehensive review. AAPS J. 7, E61–E77 (2005).
Bulcha, J. T., Wang, Y., Ma, H., Tai, P. W. L. & Gao, G. Viral vector platforms within the gene therapy landscape. Signal. Transduct. Target. Ther. 6, 53 (2021).
Mancheño-Corvo, P. & Martín-Duque, P. Viral gene therapy. Clin. Transl. Oncol. 8, 858–867 (2006).
Selot, R. S., Jayandharan, G. R. & Hareendran, S. Developing immunologically inert adeno-associated virus (AAV) vectors for gene therapy: possibilities and limitations. Curr. Pharm. Biotechnol. 14, 1072–1082 (2013).
Iyer, A. K., Duan, Z. & Amiji, M. M. Nanodelivery systems for nucleic acid therapeutics in drug resistant tumors. Mol. Pharm. 11, 2511–2526 (2014).
Rajala, A. et al. Nanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo. Nano Lett. 14, 5257–5263 (2014).
Google Scholar
Ren, T., Song, Y. K., Zhang, G. & Liu, D. Structural basis of DOTMA for its high intravenous transfection activity in mouse. Gene Ther. 7, 764–768 (2000).
Wahane, A. et al. Role of lipid-based and polymer-based non-viral vectors in nucleic acid delivery for next-generation gene therapy. Molecules 25, 2866 (2020).
Maugeri, M. et al. Linkage between endosomal escape of LNP-mRNA and loading into EVs for transport to other cells. Nat. Commun. 10, 4333 (2019).
Google Scholar
Witzigmann, D. et al. Lipid nanoparticle technology for therapeutic gene regulation in the liver. Adv. Drug Deliv. Rev. 159, 344–363 (2020).
Piotrowski-Daspit, A. S., Kauffman, A. C., Bracaglia, L. G. & Saltzman, W. M. Polymeric vehicles for nucleic acid delivery. Adv. Drug Deliv. Rev. 156, 119–132 (2020).
Rai, R., Alwani, S. & Badea, I. Polymeric nanoparticles in gene therapy: new avenues of design and optimization for delivery applications. Polymers 11, 745 (2019).
Bertschinger, M. et al. Disassembly of polyethylenimine-DNA particles in vitro: implications for polyethylenimine-mediated DNA delivery. J. Control. Rel. 116, 96–104 (2006).
Wightman, L. et al. Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J. Gene Med. 3, 362–372 (2001).
Hoyer, J. & Neundorf, I. Peptide vectors for the nonviral delivery of nucleic acids. Acc. Chem. Res. 45, 1048–1056 (2012).
Luther, D. C. et al. Delivery of drugs, proteins, and nucleic acids using inorganic nanoparticles. Adv. Drug Deliv. Rev. 156, 188–213 (2020).
Pranatharthiharan, S., Patel, M. D., D’Souza, A. A. & Devarajan, P. V. Inorganic nanovectors for nucleic acid delivery. Drug Deliv. Transl. Res. 3, 446–470 (2013).
Mora-Raimundo, P., Lozano, D., Manzano, M. & Vallet-Regí, M. Nanoparticles to knockdown osteoporosis-related gene and promote osteogenic marker expression for osteoporosis treatment. ACS Nano 13, 5451–5464 (2019).
Hadinoto, K., Sundaresan, A. & Cheow, W. S. Lipid–polymer hybrid nanoparticles as a new generation therapeutic delivery platform: a review. Eur. J. Pharm. Biopharm. 85, 427–443 (2013).
Mitchell, M. J. et al. Engineering precision nanoparticles for drug delivery. Nat. Rev. Drug Discov. 20, 101–124 (2021).
Rauch, S. et al. mRNA-based SARS-CoV-2 vaccine candidate CVnCoV induces high levels of virus-neutralising antibodies and mediates protection in rodents. NPJ Vaccines 6, 57 (2021).
Gebre, M. S. et al. Optimization of non-coding regions improves protective efficacy of an mRNA SARS-CoV-2 vaccine in nonhuman primates. Preprint at bioRxiv https://doi.org/10.1101/2021.08.13.456316 (2021).
Google Scholar
Adir, O. et al. Integrating artificial intelligence and nanotechnology for precision cancer medicine. Adv. Mater. 32, 1901989 (2020).
Hou, X., Zaks, T., Langer, R. & Dong, Y. Lipid nanoparticles for mRNA delivery. Nat. Rev. Mater. 6, 1078–1094 (2021).
Google Scholar
Akinc, A. et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat. Nanotechnol. 14, 1084–1087 (2019).
Google Scholar
[No authors listed] Let’s talk about lipid nanoparticles. Nat. Rev. Mater. 6, 99 (2021).
Mukalel, A. J., Riley, R. S., Zhang, R. & Mitchell, M. J. Nanoparticles for nucleic acid delivery: applications in cancer immunotherapy. Cancer Lett. 458, 102–112 (2019).
Nagayasu, A., Uchiyama, K. & Kiwada, H. The size of liposomes: a factor which affects their targeting efficiency to tumors and therapeutic activity of liposomal antitumor drugs. Adv. Drug Deliv. Rev. 40, 75–87 (1999).
Li, W., Huang, Z., MacKay, J. A., Grube, S. & Szoka, F. C. Jr Low-pH-sensitive poly(ethylene glycol) (PEG)-stabilized plasmid nanolipoparticles: effects of PEG chain length, lipid composition and assembly conditions on gene delivery. J. Gene Med. 7, 67–79 (2005).
Nordling-David, M. M. & Golomb, G. Gene delivery by liposomes. Isr. J. Chem. 53, 737–747 (2013).
Han, X. et al. An ionizable lipid toolbox for RNA delivery. Nat. Commun. 12, 7233 (2021).
Google Scholar
Kulkarni, J. A., Witzigmann, D., Leung, J., Tam, Y. Y. C. & Cullis, P. R. On the role of helper lipids in lipid nanoparticle formulations of siRNA. Nanoscale 11, 21733–21739 (2019).
Ambegia, E. et al. Stabilized plasmid–lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim. Biophys. Act 1669, 155–163 (2005).
Dai, Q., Walkey, C. & Chan, W. C. W. Polyethylene glycol backfilling mitigates the negative impact of the protein corona on nanoparticle cell targeting. Angew. Chem. Int. Ed. 53, 5093–5096 (2014).
Belliveau, N. M. et al. Microfluidic synthesis of highly potent limit-size lipid nanoparticles for in vivo delivery of siRNA. Mol. Ther. Nucleic Acids 1, e37 (2012).
Chen, S. et al. Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA. J. Control. Rel. 235, 236–244 (2016).
Google Scholar
Han, X., Mitchell, M. J. & Nie, G. Nanomaterials for therapeutic RNA delivery. Matter 3, 1948–1975 (2020).
Kowalski, P. S., Rudra, A., Miao, L. & Anderson, D. G. Delivering the messenger: advances in technologies for therapeutic mRNA delivery. Mol. Ther. 27, 710–728 (2019).
Kauffman, K. J. et al. Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs. Nano Lett. 15, 7300–7306 (2015).
Google Scholar
Ramaswamy, S. et al. Systemic delivery of factor IX messenger RNA for protein replacement therapy. Proc. Natl Acad. Sci. USA 114, E1941–E1950 (2017).
Byrne, J. D., Betancourt, T. & Brannon-Peppas, L. Active targeting schemes for nanoparticle systems in cancer therapeutics. Adv. Drug Deliv. Rev. 60, 1615–1626 (2008).
Jahn, A., Vreeland, W. N., Gaitan, M. & Locascio, L. E. Controlled vesicle self-assembly in microfluidic channels with hydrodynamic focusing. J. Am. Chem. Soc. 126, 2674–2675 (2004).
Zhigaltsev, I. V. et al. Bottom-up design and synthesis of limit size lipid nanoparticle systems with aqueous and triglyceride cores using millisecond microfluidic mixing. Langmuir 28, 3633–3640 (2012).
Roces, C. B. et al. Manufacturing considerations for the development of lipid nanoparticles using microfluidics. Pharmaceutics 12, 1095 (2020).
Lächelt, U. & Wagner, E. Nucleic acid therapeutics using polyplexes: a journey of 50 years (and beyond). Chem. Rev. 115, 11043–11078 (2015).
Discher, D. E. & Ahmed, F. Polymersomes. Annu. Rev. Biomed. Eng. 8, 323–341 (2006).
Iqbal, S., Blenner, M., Alexander-Bryant, A. & Larsen, J. Polymersomes for therapeutic delivery of protein and nucleic acid macromolecules: from design to therapeutic applications. Biomacromolecules 21, 1327–1350 (2020).
Pangburn, T. O., Georgiou, K., Bates, F. S. & Kokkoli, E. Targeted polymersome delivery of siRNA induces cell death of breast cancer cells dependent upon orai3 protein expression. Langmuir 28, 12816–12830 (2012).
Konishcheva, E. V., Zhumaev, U. E. & Meier, W. P. PEO-b-PCL-b-PMOXA triblock copolymers: from synthesis to microscale polymersomes with asymmetric membrane. Macromolecules 50, 1512–1520 (2017).
Google Scholar
Li, S. et al. Biodegradable polymersomes with an ionizable membrane: facile preparation, superior protein loading, and endosomal pH-responsive protein release. Eur. J. Pharm. Biopharm. 82, 103–111 (2012).
Google Scholar
Walter, M. V. & Malkoch, M. Simplifying the synthesis of dendrimers: accelerated approaches. Chem. Soc. Rev. 41, 4593–4609 (2012).
Conde, J., Oliva, N., Atilano, M., Song, H. S. & Artzi, N. Self-assembled RNA-triple-helix hydrogel scaffold for microRNA modulation in the tumour microenvironment. Nat. Mater. 15, 353–363 (2016).
Google Scholar
Palmerston Mendes, L., Pan, J. & Torchilin, V. P. Dendrimers as nanocarriers for nucleic acid and drug delivery in cancer therapy. Molecules 22, 1401 (2017).
Pack, D. W., Hoffman, A. S., Pun, S. & Stayton, P. S. Design and development of polymers for gene delivery. Nat. Rev. Drug Discov. 4, 581–593 (2005).
Lai, W.-F. & Wong, W.-T. Design of polymeric gene carriers for effective intracellular delivery. Trends Biotechnol. 36, 713–728 (2018).
Arami, H., Khandhar, A., Liggitt, D. & Krishnan, K. M. In vivo delivery, pharmacokinetics, biodistribution and toxicity of iron oxide nanoparticles. Chem. Soc. Rev. 44, 8576–8607 (2015).
Dobrovolskaia, M. A. & McNeil, S. E. Immunological properties of engineered nanomaterials. Nat. Nanotechnol. 2, 469–478 (2007).
Google Scholar
Soenen, S. J. et al. Cellular toxicity of inorganic nanoparticles: common aspects and guidelines for improved nanotoxicity evaluation. Nano Today 6, 446–465 (2011).
Cutler, J. I., Auyeung, E. & Mirkin, C. A. Spherical nucleic acids. J. Am. Chem. Soc. 134, 1376–1391 (2012).
Conde, J. et al. Dual targeted immunotherapy via in vivo delivery of biohybrid RNAi–peptide nanoparticles to tumor-associated macrophages and cancer cells. Adv. Funct. Mater. 25, 4183–4194 (2015).
Conde, J., Oliva, N. & Artzi, N. Implantable hydrogel embedded dark-gold nanoswitch as a theranostic probe to sense and overcome cancer multidrug resistance. Proc. Natl Acad. Sci. USA 112, E1278–E1287 (2015).
Google Scholar
Gilam, A. et al. Local microRNA delivery targets Palladin and prevents metastatic breast cancer. Nat. Commun. 7, 12868 (2016).
Google Scholar
Bishop, C. J., Tzeng, S. Y. & Green, J. J. Degradable polymer-coated gold nanoparticles for co-delivery of DNA and siRNA. Acta Biomater. 11, 393–403 (2015).
Donahue, N. D., Acar, H. & Wilhelm, S. Concepts of nanoparticle cellular uptake, intracellular trafficking, and kinetics in nanomedicine. Adv. Drug Deliv. Rev. 143, 68–96 (2019).
Hak, S. et al. The effect of nanoparticle polyethylene glycol surface density on ligand-directed tumor targeting studied in vivo by dual modality imaging. ACS Nano 6, 5648–5658 (2012).
Tang, S., Huang, X., Chen, X. & Zheng, N. Hollow mesoporous zirconia nanocapsules for drug delivery. Adv. Funct. Mater. 20, 2442–2447 (2010).
Ahmadi, E., Dehghannejad, N., Hashemikia, S., Ghasemnejad, M. & Tabebordbar, H. Synthesis and surface modification of mesoporous silica nanoparticles and its application as carriers for sustained drug delivery. Drug Deliv. 21, 164–172 (2014).
Li, J., Xue, S. & Mao, Z.-W. Nanoparticle delivery systems for siRNA-based therapeutics. J. Mater. Chem. B 4, 6620–6639 (2016).
Jiang, S., Eltoukhy, A. A., Love, K. T., Langer, R. & Anderson, D. G. Lipidoid-coated iron oxide nanoparticles for efficient DNA and siRNA delivery. Nano Lett. 13, 1059–1064 (2013).
Google Scholar
Tavitian, B. et al. In vivo imaging of oligonucleotides with positron emission tomography. Nat. Med. 4, 467–471 (1998).
Khorkova, O. & Wahlestedt, C. Oligonucleotide therapies for disorders of the nervous system. Nat. Biotechnol. 35, 249–263 (2017).
Kanasty, R., Dorkin, J. R., Vegas, A. & Anderson, D. Delivery materials for siRNA therapeutics. Nat. Mater. 12, 967–977 (2013).
Google Scholar
Khvorova, A. & Watts, J. K. The chemical evolution of oligonucleotide therapies of clinical utility. Nat. Biotechnol. 35, 238–248 (2017).
Dowdy, S. F. Overcoming cellular barriers for RNA therapeutics. Nat. Biotechnol. 35, 222–229 (2017).
Sun, Y. et al. Enhancing the therapeutic delivery of oligonucleotides by chemical modification and nanoparticle encapsulation. Molecules 22, 1724 (2017).
Sipa, K. et al. Effect of base modifications on structure, thermodynamic stability, and gene silencing activity of short interfering RNA. RNA 13, 1301–1316 (2007).
Orlandini von Niessen, A. G. et al. Improving mRNA-based therapeutic gene delivery by expression-augmenting 3′ UTRs identified by cellular library screening. Mol. Ther. 27, 824–836 (2019).
Holtkamp, S. et al. Modification of antigen-encoding RNA increases stability, translational efficacy, and T-cell stimulatory capacity of dendritic cells. Blood 108, 4009–4017 (2006).
Jasinski, D. L., Li, H. & Guo, P. The effect of size and shape of RNA nanoparticles on biodistribution. Mol. Ther. 26, 784–792 (2018).
Albanese, A., Tang, P. S. & Chan, W. C. W. The effect of nanoparticle size, shape, and surface chemistry on biological systems. Annu. Rev. Biomed. Eng. 14, 1–16 (2012).
Buriak, J. M. Preface to the special issue on methods and protocols in materials chemistry. Chem. Mater. 29, 1–2 (2017).
Behzadi, S. et al. Cellular uptake of nanoparticles: journey inside the cell. Chem. Soc. Rev. 46, 4218–4244 (2017).
Hadjidemetriou, M. & Kostarelos, K. Evolution of the nanoparticle corona. Nat. Nanotechnol. 12, 288–290 (2017).
Google Scholar
Satzer, P., Svec, F., Sekot, G. & Jungbauer, A. Protein adsorption onto nanoparticles induces conformational changes: particle size dependency, kinetics, and mechanisms. Eng. Life Sci. 16, 238–246 (2016).
Walkey, C. D., Olsen, J. B., Guo, H., Emili, A. & Chan, W. C. W. Nanoparticle size and surface chemistry determine serum protein adsorption and macrophage uptake. J. Am. Chem. Soc. 134, 2139–2147 (2012).
Glancy, D. et al. Characterizing the protein corona of sub-10 nm nanoparticles. J. Control. Rel. 304, 102–110 (2019).
Faria, M. et al. Minimum information reporting in bio–nano experimental literature. Nat. Nanotechnol. 13, 777–785 (2018).
Google Scholar
Modena, M. M., Rühle, B., Burg, T. P. & Wuttke, S. Nanoparticle characterization: what to measure? Adv. Mater. 31, 1901556 (2019).
Varenne, F., Makky, A., Gaucher-Delmas, M., Violleau, F. & Vauthier, C. Multimodal dispersion of nanoparticles: a comprehensive evaluation of size distribution with 9 size measurement methods. Pharm. Res. 33, 1220–1234 (2016).
Stetefeld, J., McKenna, S. A. & Patel, T. R. Dynamic light scattering: a practical guide and applications in biomedical sciences. Biophys. Rev. 8, 409–427 (2016).
Shen, H. et al. Single particle tracking: from theory to biophysical applications. Chem. Rev. 117, 7331–7376 (2017).
Dahmen, U. et al. Background, status and future of the transmission electron aberration-corrected microscope project. Philos. Trans. R. Soc. A Math. Phys. Eng. Sci. 367, 3795–3808 (2009).
Google Scholar
Skowron, S. T. et al. Chemical reactions of molecules promoted and simultaneously imaged by the electron beam in transmission electron microscopy. Acc. Chem. Res. 50, 1797–1807 (2017).
Nakamura, E., Sommerdijk, N. A. J. M. & Zheng, H. Transmission electron microscopy for chemists. Acc. Chem. Res. 50, 1795–1796 (2017).
Stewart, P. L. Cryo-electron microscopy and cryo-electron tomography of nanoparticles. WIREs Nanomed. Nanobiotechnol. 9, e1417 (2017).
Mourdikoudis, S., Pallares, R. M. & Thanh, N. T. K. Characterization techniques for nanoparticles: comparison and complementarity upon studying nanoparticle properties. Nanoscale 10, 12871–12934 (2018).
Rao, A. et al. Characterization of nanoparticles using atomic force microscopy. J. Phys. Conf. Ser. 61, 971–976 (2007).
Google Scholar
Glass, J. J. et al. Charge has a marked influence on hyperbranched polymer nanoparticle association in whole human blood. ACS Macro Lett. 6, 586–592 (2017).
Salatin, S., Maleki Dizaj, S. & Yari Khosroushahi, A. Effect of the surface modification, size, and shape on cellular uptake of nanoparticles. Cell Biol. Int. 39, 881–890 (2015).
Doane, T. L., Chuang, C.-H., Hill, R. J. & Burda, C. Nanoparticle ζ-potentials. Acc. Chem. Res. 45, 317–326 (2012).
Mura, S., Nicolas, J. & Couvreur, P. Stimuli-responsive nanocarriers for drug delivery. Nat. Mater. 12, 991–1003 (2013).
Google Scholar
Colombo, M. et al. Tumour homing and therapeutic effect of colloidal nanoparticles depend on the number of attached antibodies. Nat. Commun. 7, 13818 (2016).
Google Scholar
Cheng, Y. et al. A multifunctional peptide-conjugated AIEgen for efficient and sequential targeted gene delivery into the nucleus. Angew. Chem. Int. Ed. 58, 5049–5053 (2019).
Herda, L. M., Hristov, D. R., Lo Giudice, M. C., Polo, E. & Dawson, K. A. Mapping of molecular structure of the nanoscale surface in bionanoparticles. J. Am. Chem. Soc. 139, 111–114 (2017).
Conte, C. et al. Multi-component bioresponsive nanoparticles for synchronous delivery of docetaxel and TUBB3 siRNA to lung cancer cells. Nanoscale 13, 11414–11426 (2021).
Chen, F. et al. Complement proteins bind to nanoparticle protein corona and undergo dynamic exchange in vivo. Nat. Nanotechnol. 12, 387–393 (2017).
Google Scholar
Kim, J., Koo, B.-K. & Knoblich, J. A. Human organoids: model systems for human biology and medicine. Nat. Rev. Mol. Cell Biol. 21, 571–584 (2020).
Skylar-Scott, M. A. et al. Biomanufacturing of organ-specific tissues with high cellular density and embedded vascular channels. Sci. Adv. 5, eaaw2459 (2022).
Google Scholar
Low, L. A., Mummery, C., Berridge, B. R., Austin, C. P. & Tagle, D. A. Organs-on-chips: into the next decade. Nat. Rev. Drug Discov. 20, 345–361 (2021).
Kohl, Y. et al. Microfluidic in vitro platform for (nano)safety and (nano)drug efficiency screening. Small 17, 2006012 (2021).
Conde, J., Oliva, N., Zhang, Y. & Artzi, N. Local triple-combination therapy results in tumour regression and prevents recurrence in a colon cancer model. Nat. Mater. 15, 1128–1138 (2016).
Google Scholar
Poley, M. et al. Chemotherapeutic nanoparticles accumulate in the female reproductive system during ovulation affecting fertility and anticancer activity. Preprint at bioRxiv https://doi.org/10.1101/2020.07.22.216168 (2020).
Google Scholar
Duan, X. & Li, Y. Physicochemical characteristics of nanoparticles affect circulation, biodistribution, cellular internalization, and trafficking. Small 9, 1521–1532 (2013).
Elci, S. G. et al. Surface charge controls the suborgan biodistributions of gold nanoparticles. ACS Nano 10, 5536–5542 (2016).
Soo Choi, H. et al. Renal clearance of quantum dots. Nat. Biotechnol. 25, 1165–1170 (2007).
Hoshyar, N., Gray, S., Han, H. & Bao, G. The effect of nanoparticle size on in vivo pharmacokinetics and cellular interaction. Nanomedicine 11, 673–692 (2016).
García, K. P. et al. Zwitterionic-coated “stealth” nanoparticles for biomedical applications: recent advances in countering biomolecular corona formation and uptake by the mononuclear phagocyte system. Small 10, 2516–2529 (2014).
Moitra, P., Alafeef, M., Dighe, K., Frieman, M. B. & Pan, D. Selective naked-eye detection of SARS-CoV-2 mediated by N gene targeted antisense oligonucleotide capped plasmonic nanoparticles. ACS Nano 14, 7617–7627 (2020).
Jwa-Min, N., Shad, T. C. & Mirkin, C. A. Nanoparticle-based bio-bar codes for the ultrasensitive detection of proteins. Science 301, 1884–1886 (2003).
Google Scholar
Liu, J. & Lu, Y. Fast colorimetric sensing of adenosine and cocaine based on a general sensor design involving aptamers and nanoparticles. Angew. Chem. Int. Ed. 45, 90–94 (2006).
Liu, J. & Lu, Y. A colorimetric lead biosensor using DNAzyme-directed assembly of gold nanoparticles. J. Am. Chem. Soc. 125, 6642–6643 (2003).
Robert, E., Storhoff, J. J., Mucic, R. C., Letsinger, R. L. & Mirkin, C. A. Selective colorimetric detection of polynucleotides based on the distance-dependent optical properties of gold nanoparticles. Science 277, 1078–1081 (1997).
Andrew, T. T., Mirkin, C. A. & Letsinger, R. L. Scanometric DNA array detection with nanoparticle probes. Science 289, 1757–1760 (2000).
Google Scholar
Charles, C. Y., Rongchao, J. & Mirkin, C. A. Nanoparticles with Raman spectroscopic fingerprints for DNA and RNA detection. Science 297, 1536–1540 (2002).
Google Scholar
Walker, T. et al. Clinical Impact of laboratory implementation of verigene BC-GN microarray-based assay for detection of Gram-negative bacteria in positive blood cultures. J. Clin. Microbiol. 54, 1789–1796 (2016).
Georganopoulou, D. G. et al. Nanoparticle-based detection in cerebral spinal fluid of a soluble pathogenic biomarker for Alzheimer’s disease. Proc. Natl Acad. Sci. USA 102, 2273–2276 (2005).
Google Scholar
Han, M. S., Lytton-Jean, A. K. R., Oh, B.-K., Heo, J. & Mirkin, C. A. Colorimetric screening of DNA-binding molecules with gold nanoparticle probes. Angew. Chem. Int. Ed. 45, 1807–1810 (2006).
Liu, J. & Lu, Y. Accelerated color change of gold nanoparticles assembled by DNAzymes for simple and fast colorimetric Pb2+ detection. J. Am. Chem. Soc. 126, 12298–12305 (2004).
Yang, L. et al. Efficient delivery of antisense oligonucleotides using bioreducible lipid nanoparticles in vitro and in vivo. Mol. Ther. Nucleic Acids 19, 1357–1367 (2020).
Khan, F. O. et al. Endothelial siRNA delivery in nonhuman primates using ionizable low-molecular weight polymeric nanoparticles. Sci. Adv. 4, eaar8409 (2021).
Google Scholar
Wei, T. et al. siRNA nanoparticles targeting CaMKIIγ in lesional macrophages improve atherosclerotic plaque stability in mice. Sci. Transl. Med. 12, eaay1063 (2020).
Conde, J. et al. Design of multifunctional gold nanoparticles for in vitro and in vivo gene silencing. ACS Nano 6, 8316–8324 (2012).
Wen, L. et al. BBB pathophysiology-independent delivery of siRNA in traumatic brain injury. Sci. Adv. 7, eabd6889 (2021).
Google Scholar
Zhao, W., Hou, X., Vick, O. G. & Dong, Y. RNA delivery biomaterials for the treatment of genetic and rare diseases. Biomaterials 217, 119291 (2019).
Zimmermann, T. S. et al. RNAi-mediated gene silencing in non-human primates. Nature 441, 111–114 (2006).
Google Scholar
Morrissey, D. V. et al. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat. Biotechnol. 23, 1002–1007 (2005).
Mendes, B. B., Sousa, D. P., Conniot, J. & Conde, J. Nanomedicine-based strategies to target and modulate the tumor microenvironment. Trends Cancer 7, 847–862 (2021).
Woodrow, K. A. et al. Intravaginal gene silencing using biodegradable polymer nanoparticles densely loaded with small-interfering RNA. Nat. Mater. 8, 526–533 (2009).
Google Scholar
Wilson, D. S. et al. Orally delivered thioketal nanoparticles loaded with TNF-α–siRNA target inflammation and inhibit gene expression in the intestines. Nat. Mater. 9, 923–928 (2010).
Google Scholar
Debacker, A. J., Voutila, J., Catley, M., Blakey, D. & Habib, N. Delivery of oligonucleotides to the liver with GalNAc: from research to registered therapeutic drug. Mol. Ther. 28, 1759–1771 (2020).
Zuris, J. A. et al. Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo. Nat. Biotechnol. 33, 73–80 (2015).
Zhang, H.-X., Zhang, Y. & Yin, H. Genome editing with mRNA encoding ZFN, TALEN, and Cas9. Mol. Ther. 27, 735–746 (2019).
Xu, C.-F. et al. Rational designs of in vivo CRISPR–Cas delivery systems. Adv. Drug Deliv. Rev. 168, 3–29 (2021).
Liu, S. et al. Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR–Cas gene editing. Nat. Mater. 20, 701–710 (2021).
Google Scholar
Parayath, N. N., Stephan, S. B., Koehne, A. L., Nelson, P. S. & Stephan, M. T. In vitro-transcribed antigen receptor mRNA nanocarriers for transient expression in circulating T cells in vivo. Nat. Commun. 11, 6080 (2020).
Google Scholar
Cheng, Q. et al. Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing. Nat. Nanotechnol. 15, 313–320 (2020).
Google Scholar
Wolff, A. J. et al. Direct gene transfer into mouse muscle in vivo. Science 247, 1465–1468 (1990).
Google Scholar
Sabnis, S. et al. A novel amino lipid series for mRNA delivery: improved endosomal escape and sustained pharmacology and safety in non-human primates. Mol. Ther. 26, 1509–1519 (2018).
Islam, M. A. et al. Restoration of tumour-growth suppression in vivo via systemic nanoparticle-mediated delivery of PTEN mRNA. Nat. Biomed. Eng. 2, 850–864 (2018).
Kong, N. et al. Synthetic mRNA nanoparticle-mediated restoration of p53 tumor suppressor sensitizes p53-deficient cancers to mTOR inhibition. Sci. Transl. Med. 11, eaaw1565 (2019).
Yao-Xin, L. et al. Reactivation of the tumor suppressor PTEN by mRNA nanoparticles enhances antitumor immunity in preclinical models. Sci. Transl. Med. 13, eaba9772 (2021).
Chaudhary, N., Weissman, D. & Whitehead, K. A. mRNA vaccines for infectious diseases: principles, delivery and clinical translation. Nat. Rev. Drug Discov. 20, 817–838 (2021).
Pardi, N., Hogan, M. J., Porter, F. W. & Weissman, D. mRNA vaccines — a new era in vaccinology. Nat. Rev. Drug Discov. 17, 261–279 (2018).
Islam, M. A. et al. Adjuvant-pulsed mRNA vaccine nanoparticle for immunoprophylactic and therapeutic tumor suppression in mice. Biomaterials 266, 120431 (2021).
Zhang, H. et al. Delivery of mRNA vaccine with a lipid-like material potentiates antitumor efficacy through Toll-like receptor 4 signaling. Proc. Natl Acad. Sci. USA 118, e2005191118 (2021).
Smith, T. T. et al. In situ programming of leukaemia-specific T cells using synthetic DNA nanocarriers. Nat. Nanotechnol. 12, 813–820 (2017).
Capece, D., Verzella, D., Fischietti, M., Zazzeroni, F. & Alesse, E. Targeting costimulatory molecules to improve antitumor immunity. J. Biomed. Biotechnol. 2012, 926321 (2012).
Hewitt, L. S. et al. Durable anticancer immunity from intratumoral administration of IL-23, IL-36γ, and OX40L mRNAs. Sci. Transl. Med. 11, eaat9143 (2019).
Conniot, J. et al. Immunization with mannosylated nanovaccines and inhibition of the immune-suppressing microenvironment sensitizes melanoma to immune checkpoint modulators. Nat. Nanotechnol. 14, 891–901 (2019).
Google Scholar
Sago, C. D. et al. High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing. Proc. Natl Acad. Sci. USA 115, E9944–E9952 (2018).
Guimaraes, P. P. G. et al. Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening. J. Control. Rel. 316, 404–417 (2019).
Seiichi, O., Dylan, G. & Chan, W. C. W. DNA-controlled dynamic colloidal nanoparticle systems for mediating cellular interaction. Science 351, 841–845 (2016).
Schrurs, F. & Lison, D. Focusing the research efforts. Nat. Nanotechnol. 7, 546–548 (2012).
Google Scholar
Cheng, Y., Morshed, R. A., Auffinger, B., Tobias, A. L. & Lesniak, M. S. Multifunctional nanoparticles for brain tumor imaging and therapy. Adv. Drug Deliv. Rev. 66, 42–57 (2014).
Jain, R. K. & Stylianopoulos, T. Delivering nanomedicine to solid tumors. Nat. Rev. Clin. Oncol. 7, 653–664 (2010).
Junttila, M. R. & de Sauvage, F. J. Influence of tumour micro-environment heterogeneity on therapeutic response. Nature 501, 346–354 (2013).
Google Scholar
Bedard, P. L., Hansen, A. R., Ratain, M. J. & Siu, L. L. Tumour heterogeneity in the clinic. Nature 501, 355–364 (2013).
Google Scholar
Leong, H. S. et al. On the issue of transparency and reproducibility in nanomedicine. Nat. Nanotechnol. 14, 629–635 (2019).
Google Scholar
Lammers, T. & Storm, G. Setting standards to promote progress in bio–nano science. Nat. Nanotechnol. 14, 626 (2019).
Google Scholar
Florindo, H. F., Madi, A. & Satchi-Fainaro, R. Challenges in the implementation of MIRIBEL criteria on nanobiomed manuscripts. Nat. Nanotechnol. 14, 627–628 (2019).
Google Scholar
[No authors listed] Voices from the community. Nat. Nanotechnol. 14, 625 (2019).
Google Scholar
Zu, H. & Gao, D. Non-viral vectors in gene therapy: recent development, challenges, and prospects. AAPS J. 23, 78 (2021).
Hill, A. B., Chen, M., Chen, C.-K., Pfeifer, B. A. & Jones, C. H. Overcoming gene-delivery hurdles: physiological considerations for nonviral vectors. Trends Biotechnol. 34, 91–105 (2016).
Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M. & Abedi, M. R. Gene therapy clinical trials worldwide to 2017: an update. J. Gene Med. 20, e3015 (2018).
Ramamoorth, M. & Narvekar, A. Non viral vectors in gene therapy — an overview. J. Clin. Diagn. Res. 9, GE01–GE06 (2015).
Wei, Y., Quan, L., Zhou, C. & Zhan, Q. Factors relating to the biodistribution & clearance of nanoparticles & their effects on in vivo application. Nanomedicine 13, 1495–1512 (2018).
Sato, Y. et al. Elucidation of the physicochemical properties and potency of siRNA-loaded small-sized lipid nanoparticles for siRNA delivery. J. Control. Rel. 229, 48–57 (2016).
Cabral, H. et al. Accumulation of sub-100 nm polymeric micelles in poorly permeable tumours depends on size. Nat. Nanotechnol. 6, 815–823 (2011).
Google Scholar
Phan, H. T. & Haes, A. J. What does nanoparticle stability mean? J. Phys. Chem. C 123, 16495–16507 (2019).
Roberts, T. C., Langer, R. & Wood, M. J. A. Advances in oligonucleotide drug delivery. Nat. Rev. Drug Discov. 19, 673–694 (2020).
Lorenc, A. et al. Machine learning for next-generation nanotechnology in healthcare. Matter 4, 3078–3080 (2021).
Shi, Y., van der Meel, R., Chen, X. & Lammers, T. The EPR effect and beyond: strategies to improve tumor targeting and cancer nanomedicine treatment efficacy. Theranostics 10, 7921–7924 (2020).
Park, J. et al. Alliance with EPR effect: combined strategies to improve the EPR effect in the tumor microenvironment. Theranostics 9, 8073–8090 (2019).
Paunovska, K. et al. Analyzing 2000 in vivo drug delivery data points reveals cholesterol structure impacts nanoparticle delivery. ACS Nano 12, 8341–8349 (2018).
Vaughan, H. J., Green, J. J. & Tzeng, S. Y. Cancer-targeting nanoparticles for combinatorial nucleic acid delivery. Adv. Mater. 32, 1901081 (2020).
Chakraborty, C., Sharma, A. R., Bhattacharya, M. & Lee, S.-S. From COVID-19 to cancer mRNA vaccines: moving from bench to clinic in the vaccine landscape. Front. Immunol. 12, 2648 (2021).
Tombácz, I., Weissman, D. & Pardi, N. in Vaccination with Messenger RNA: A Promising Alternative to DNA Vaccination BT – DNA Vaccines: Methods and Protocols (ed. Sousa, Â.) 13–31 (Springer US, 2021).
Maruggi, G., Zhang, C., Li, J., Ulmer, J. B. & Yu, D. mRNA as a transformative technology for vaccine development to control infectious diseases. Mol. Ther. 27, 757–772 (2019).
Fundytus, A. et al. Access to cancer medicines deemed essential by oncologists in 82 countries: an international, cross-sectional survey. Lancet Oncol. 22, 1367–1377 (2021).
Excler, J.-L., Privor-Dumm, L. & Kim, J. H. Supply and delivery of vaccines for global health. Curr. Opin. Immunol. 71, 13–20 (2021).
Eccleston-Turner, M. & Upton, H. International collaboration to ensure equitable access to vaccines for COVID-19: the ACT-Accelerator and the COVAX Facility. Milbank Q. 99, 426–449 (2021).
Plotkin, S., Robinson, J. M., Cunningham, G., Iqbal, R. & Larsen, S. The complexity and cost of vaccine manufacturing — an overview. Vaccine 35, 4064–4071 (2017).
Prager, F. D. Historic background and foundation of american patent law. Am. J. Leg. Hist. 5, 309–325 (1961).
Lévêque, F. & Ménière, Y. Patents and innovation: friends or foes? SSRN https://doi.org/10.2139/ssrn.958830 (2006).
Google Scholar
Braithwaite, J. Corporate Crime in the Pharmaceutical Industry (Routledge Revivals) (Routledge, 2013).
Okereke, M. Towards vaccine equity: should big pharma waive intellectual property rights for COVID-19 vaccines? Public Heal. Pract. 2, 100165 (2021).
Kon, E., Elia, U. & Peer, D. Principles for designing an optimal mRNA lipid nanoparticle vaccine. Curr. Opin. Biotechnol. 73, 329–336 (2022).
Ramishetti, S. et al. A combinatorial library of lipid nanoparticles for RNA delivery to leukocytes. Adv. Mater. 32, 1906128 (2020).
Billingsley, M. M. et al. Ionizable lipid nanoparticle-mediated mRNA delivery for human CAR T cell engineering. Nano Lett. 20, 1578–1589 (2020).
Google Scholar
Yamankurt, G. et al. Exploration of the nanomedicine-design space with high-throughput screening and machine learning. Nat. Biomed. Eng. 3, 318–327 (2019).
Shamay, Y. et al. Quantitative self-assembly prediction yields targeted nanomedicines. Nat. Mater. 17, 361–368 (2018).
Google Scholar
Yesselman, J. D. et al. Computational design of three-dimensional RNA structure and function. Nat. Nanotechnol. 14, 866–873 (2019).
Google Scholar
Leppek, K. et al. Combinatorial optimization of mRNA structure, stability, and translation for RNA-based therapeutics. Preprint at bioRxiv https://doi.org/10.1101/2021.03.29.437587 (2021).
Google Scholar
