Chaudhary, N., Weissman, D. & Whitehead, K. A. mRNA vaccines for infectious diseases: principles, delivery and clinical translation. Nat. Rev. Drug Discov. 20, 817 (2021).
Pardi, N., Hogan, M. J., Porter, F. W. & Weissman, D. mRNA vaccines—a new era in vaccinology. Nat. Rev. Drug Discov. 17, 261 (2018).
Sahin, U., Karikó, K. & Türeci, O. mRNA-based therapeutics—developing a new class of drugs. Nat. Rev. Drug Discov. 13, 759 (2014).
Mendes, B. B. et al. Nanodelivery of nucleic acids. Nat. Rev. Methods Prim. 2, 24 (2022).
Pastor, F. et al. An RNA toolbox for cancer immunotherapy. Nat. Rev. Drug Discov. 17, 751 (2018).
Miao, L., Zhang, Y. & Huang, L. mRNA vaccine for cancer immunotherapy. Mol. Cancer 20, 41 (2021).
Zhang, H., Zhang, Y. & Yin, H. Genome editing with mRNA encoding ZFN, TALEN, and Cas9. Mol. Ther. 27, 735 (2019).
Yin, H., Kauffman, K. J. & Anderson, D. G. Delivery technologies for genome editing. Nat. Rev. Drug Discov. 16, 387 (2017).
Akinc, A. et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat. Nanotechnol. 14, 1084 (2019).
Hou, X., Zaks, T., Langer, R. & Dong, Y. Lipid nanoparticles for mRNA delivery. Nat. Rev. Mater. 6, 1078 (2021).
Hajj, K. A. & Whitehead, K. A. Tools for translation: non-viral materials for therapeutic mRNA delivery. Nat. Rev. Mater. 2, 17056 (2017).
Finn, J. D. et al. A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep. 22, 2227 (2018).
Gillmore, J. D. et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N. Engl. J. Med. 385, 493 (2021).
Whitehead, K. A. et al. Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity. Nat. Commun. 5, 4277 (2014).
Qiu, M., Li, Y., Bloomer, H. & Xu, Q. Developing biodegradable lipid nanoparticles for intracellular mRNA delivery and genome editing. Acc. Chem. Res. 54, 4001 (2021).
Hou, X. et al. Vitamin lipid nanoparticles enable adoptive macrophage transfer for the treatment of multidrug-resistant bacterial sepsis. Nat. Nanotechnol. 15, 41 (2020).
Zhao, X. et al. Imidazole-based synthetic lipidoids for in vivo mRNA delivery into primary T lymphocytes. Angew. Chem. Int. Ed. 59, 20083 (2020).
Zhou, K. et al. Modular degradable dendrimers enable small RNAs to extend survival in an aggressive liver cancer model. Proc. Natl Acad. Sci. USA 113, 520 (2016).
Miao, L. et al. Delivery of mRNA vaccines with heterocyclic lipids increases anti-tumor efficacy by STING-mediated immune cell activation. Nat. Biotechnol. 37, 1174 (2019).
Xue, L. et al. Rational design of bisphosphonate lipid-like materials for mRNA delivery to the bone microenvironment. J. Am. Chem. Soc. 144, 9926 (2022).
Li, W. et al. Biomimetic nanoparticles deliver mRNAs encoding costimulatory receptors and enhance T cell mediated cancer immunotherapy. Nat. Commun. 12, 7264 (2021).
Cheng, Q. et al. Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing. Nat. Nanotechnol. 15, 313 (2020).
Kulkarni, J. A., Witzigmann, D., Chen, S., Cullis, P. R. & van der Meel, R. Lipid nanoparticle technology for clinical translation of siRNA therapeutics. Acc. Chem. Res. 52, 2435 (2019).
Liu, S. et al. Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR-Cas gene editing. Nat. Mater. 20, 701 (2021).
Dilliard, S. A., Cheng, Q. & Siegwart, D. J. On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles. Proc. Natl Acad. Sci. USA 118, e2109256118 (2021).
Qiu, M. et al. Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis. Proc. Natl Acad. Sci. USA 119, e2116271119 (2022).
Shahbazi, M. A., Herranz, B. & Santos, H. A. Nanostructured porous Si-based nanoparticles for targeted drug delivery. Biomatter 2, 296 (2012).
Tang, F., Li, L. & Chen, D. Mesoporous silica nanoparticles: synthesis, biocompatibility and drug delivery. Adv. Mater. 24, 1504 (2012).
Frampton, M. B. et al. Exploring the utility of hybrid siloxane-phosphocholine (SiPC) liposomes as drug delivery vehicles. RSC Adv. 11, 13014 (2021).
Semple, S. C. et al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol. 28, 172 (2010).
Zhu, Y. et al. Multi-step screening of DNA/lipid nanoparticles and co-delivery with siRNA to enhance and prolong gene expression. Nat. Commun. 13, 4282 (2022).
Hu, B. et al. Thermostable ionizable lipid-like nanoparticles (iLAND) for RNAi treatment of hyperlipidemia. Sci. Adv. 8, eabm1418 (2022).
Ni, X., Kelly, S. S., Xu, S. & Xian, M. The path to controlled delivery of reactive sulfur species. Acc. Chem. Res. 54, 3968 (2021).
Behzadi, S. et al. Cellular uptake of nanoparticles: journey inside the cell. Chem. Soc. Rev. 46, 4218 (2017).
Wei, Y. et al. A cationic lipid with advanced membrane fusion performance for pDNA and mRNA delivery. J. Mater. Chem. B 11, 2095 (2023).
Tokudome, Y. et al. Preparation and characterization of ceramide-based liposomes with high fusion activity and high membrane fluidity. Colloids Surf. B 73, 92 (2009).
Akinc, A. et al. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat. Biotechnol. 26, 561 (2008).
Paunovska, K. et al. A direct comparison of in vitro and in vivo nucleic acid delivery mediated by hundreds of nanoparticles reveals a weak correlation. Nano Lett. 18, 2148 (2018).
Nagy, A. Cre recombinase: the universal reagent for genome tailoring. Genesis 26, 99 (2000).
Hajj, K. A. et al. A potent branched-tail lipid nanoparticle enables multiplexed mRNA delivery and gene editing in vivo. Nano Lett. 20, 5167 (2020).
Singh, B., Fu, C. & Bhattacharya, J. Vascular expression of the αvβ3-integrin in lung and other organs. Am. J. Physiol. Lung Cell. Mol. Physiol. 278, L217 (2000).
Alton, E. et al. Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung. Gene Ther. 21, 89 (2014).
Ebos, J. & Kerbel, R. S. Antiangiogenic therapy: impact on invasion, disease progression, and metastasis. Nat. Rev. Clin. Oncol. 8, 210 (2011).
Xue, L. et al. High-throughput barcoding of nanoparticles identifies cationic, degradable lipid-like materials for mRNA delivery to the lungs in female preclinical models. Nat. Commun. 15, 1884 (2024).
Zhao, G. et al. TGF-βR2 signaling coordinates pulmonary vascular repair after viral injury in mice and human tissue. Sci. Trans. Med. 16, eadg6229 (2024).
Jia, T. et al. FGF-2 promotes angiogenesis through a SRSF1/SRSF3/SRPK1-dependent axis that controls VEGFR1 splicing in endothelial cells. BMC Biol. 19, 173 (2021).
Cao, R. et al. Comparative evaluation of FGF-2-, VEGF-A-, and VEGF-C-induced angiogenesis, lymphangiogenesis, vascular fenestrations, and permeability. Circ. Res. 94, 664 (2004).
Dahlman, J. E. et al. In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight. Nat. Nanotechnol. 9, 648 (2014).
McDermott, M. R., Brook, M. A. & Bartzoka, V. Adjuvancy effect of different types of silicone gel. J. Biomed. Mater. Res. 46, 132 (1999).
Huang, X. et al. Genome editing abrogates angiogenesis in vivo. Nat. Commun. 8, 112 (2017).
Wei, T. et al. Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing. Nat. Commun. 11, 3232 (2020).
Momany, F. & Rone, R. Validation of the general purpose QUANTA ®3.2/CHARMm® force field. J. Comput. Chem. 13, 888 (1992).