Gillmore, J. D. et al. CRISPR–Cas9 in vivo gene editing for transthyretin amyloidosis. N. Engl. J. Med. 385, 493–502 (2021).
Mendell, J. R. et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017).
Pasi, K. J. et al. Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A. N. Engl. J. Med. 382, 29–40 (2020).
Sahin, U. et al. An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma. Nature 585, 107–112 (2020).
Kulkarni, J. A. et al. The current landscape of nucleic acid therapeutics. Nat. Nanotechnol. 16, 630–643 (2021).
Yin, H. et al. Non-viral vectors for gene-based therapy. Nat. Rev. Genet. 15, 541–555 (2014).
Goldberg, M. S. Improving cancer immunotherapy through nanotechnology. Nat. Rev. Cancer 19, 587–602 (2019).
Hammond, S. M. et al. Delivery of oligonucleotide-based therapeutics: challenges and opportunities. EMBO Mol. Med. 13, e13243 (2021).
Khvorova, A. & Watts, J. K. The chemical evolution of oligonucleotide therapies of clinical utility. Nat. Biotechnol. 35, 238–248 (2017).
Mendell, J. R. et al. Current clinical applications of in vivo gene therapy with AAVs. Mol. Ther. 29, 464–488 (2021).
Zhao, Z., Ukidve, A., Kim, J. & Mitragotri, S. Targeting strategies for tissue-specific drug delivery. Cell 181, 151–167 (2020).
Vargason, A. M., Anselmo, A. C. & Mitragotri, S. The evolution of commercial drug delivery technologies. Nat. Biomed. Eng. 5, 951–967 (2021).
Dowdy, S. F. Overcoming cellular barriers for RNA therapeutics. Nat. Biotechnol. 35, 222–229 (2017).
Song, X. et al. Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy. Adv. Drug Deliv. Rev. 168, 158–180 (2021).
Paunovska, K., Loughrey, D. & Dahlman, J. E. Drug delivery systems for RNA therapeutics. Nat. Rev. Genet. 23, 265–280 (2022).
Marx, V. Cell biology: delivering tough cargo into cells. Nat. Methods 13, 37–40 (2015).
Aihara, H. & Miyazaki, J. Gene transfer into muscle by electroporation in vivo. Nat. Biotechnol. 16, 867–870 (1998).
Wilson, A. M. et al. In vivo laser-mediated retinal ganglion cell optoporation using KV1.1 conjugated gold nanoparticles. Nano Lett. 18, 6981–6988 (2018).
Carpentier, A. et al. Clinical trial of blood–brain barrier disruption by pulsed ultrasound. Sci. Transl. Med. 8, 343re342 (2016).
dal Maschio, M. et al. High-performance and site-directed in utero electroporation by a triple-electrode probe. Nat. Commun. 3, 960 (2012).
Kar, A. et al. Wearable and implantable devices for drug delivery: applications and challenges. Biomaterials 283, 121435 (2022).
van Haasteren, J., Li, J., Scheideler, O. J., Murthy, N. & Schaffer, D. V. The delivery challenge: fulfilling the promise of therapeutic genome editing. Nat. Biotechnol. 38, 845–855 (2020).
Shao, H. et al. New technologies for analysis of extracellular vesicles. Chem. Rev. 118, 1917–1950 (2018).
Wang, Q. et al. ARMMs as a versatile platform for intracellular delivery of macromolecules. Nat. Commun. 9, 960 (2018).
de Jong, O. G. et al. A CRISPR–Cas9-based reporter system for single-cell detection of extracellular vesicle-mediated functional transfer of RNA. Nat. Commun. 11, 1113 (2020).
Coccolini, F. et al. Liver trauma: WSES 2020 guidelines. World J. Emerg. Surg. 15, 24 (2020).
Kisseleva, T. & Brenner, D. Molecular and cellular mechanisms of liver fibrosis and its regression. Nat. Rev. Gastroenterol. Hepatol. 18, 151–166 (2021).
Yamakawa, S. & Hayashida, K. Advances in surgical applications of growth factors for wound healing. Burns Trauma 7, 10 (2019).
Song, Z. et al. Epidermal growth factor receptor signaling regulates β cell proliferation in adult mice. J. Biol. Chem. 291, 22630–22637 (2016).
Vogelstein, B. et al. Cancer genome landscapes. Science 339, 1546–1558 (2013).
Dentro, S. C. et al. Characterizing genetic intra-tumor heterogeneity across 2,658 human cancer genomes. Cell 184, 2239–2254 (2021).
Graham, T. A. & Sottoriva, A. Measuring cancer evolution from the genome. J. Pathol. 241, 183–191 (2017).
Liu, X. et al. Somatic loss of BRCA1 and p53 in mice induces mammary tumors with features of human BRCA1-mutated basal-like breast cancer. Proc. Natl Acad. Sci. USA 104, 12111–12116 (2007).
Husemann, Y. et al. Systemic spread is an early step in breast cancer. Cancer Cell 13, 58–68 (2008).
Attalla, S., Taifour, T., Bui, T. & Muller, W. Insights from transgenic mouse models of PyMT-induced breast cancer: recapitulating human breast cancer progression in vivo. Oncogene 40, 475–491 (2021).
Huen, M. S., Sy, S. M. & Chen, J. BRCA1 and its toolbox for the maintenance of genome integrity. Nat. Rev. Mol. Cell Biol. 11, 138–148 (2010).
Tutt, A. et al. Oral poly(ADP-ribose) polymerase inhibitor olaparib in patients with BRCA1 or BRCA2 mutations and advanced breast cancer: a proof-of-concept trial. Lancet 376, 235–244 (2010).
LaFargue, C. J., Dal Molin, G. Z., Sood, A. K. & Coleman, R. L. Exploring and comparing adverse events between PARP inhibitors. Lancet Oncol. 20, e15–e28 (2019).
Parent, M. et al. PLGA in situ implants formed by phase inversion: critical physicochemical parameters to modulate drug release. J. Control. Release 172, 292–304 (2013).
Fredenberg, S., Wahlgren, M., Reslow, M. & Axelsson, A. The mechanisms of drug release in poly(lactic-co-glycolic acid)-based drug delivery systems–a review. Int. J. Pharm. 415, 34–52 (2011).
Pagani, O. et al. International guidelines for management of metastatic breast cancer: can metastatic breast cancer be cured? J. Natl Cancer Inst. 102, 456–463 (2010).
Jin, X. & Mu, P. Targeting breast cancer metastasis. Breast Cancer 9, 23–34 (2015).
Kato, T. et al. A novel human tRNA–dihydrouridine synthase involved in pulmonary carcinogenesis. Cancer Res. 65, 5638–5646 (2005).
Xie, Z., Avila, R., Huang, Y. & Rogers, J. A. Flexible and stretchable antennas for biointegrated electronics. Adv. Mater. 32, e1902767 (2020).
Ghovanloo, M. & Najafi, K. A wideband frequency-shift keying wireless link for inductively powered biomedical implants. IEEE Trans. Circuits Syst. I 51, 2374–2383 (2004).
Li, X. et al. Lipoplex-mediated single-cell transfection via droplet microfluidics. Small 14, e1802055 (2018).
Annunziato, S. et al. Modeling invasive lobular breast carcinoma by CRISPR/Cas9-mediated somatic genome editing of the mammary gland. Genes Dev. 30, 1470–1480 (2016).
Vella, L. J. et al. A rigorous method to enrich for exosomes from brain tissue. J. Extracell. Vesicles 6, 1348885 (2017).
Mohammed, B. M., Monroe, D. M. & Gailani, D. Mouse models of hemostasis. Platelets 31, 417–422 (2020).
Li, Y. et al. Self-adapting hydrogel to improve the therapeutic effect in wound-healing. ACS Appl. Mater. Interfaces 10, 26046–26055 (2018).
Suzuki, S. et al. The beneficial effect of a prostaglandin 12 analog on ischemic rat liver. Transplantation 52, 979–983 (1991).
Tian, Y. et al. Mesenchymal stem cells improve mouse non-heart-beating liver graft survival by inhibiting Kupffer cell apoptosis via TLR4–ERK1/2–Fas/FasL–caspase3 pathway regulation. Stem Cell Res. Ther. 7, 157 (2016).
Diaz-Cruz, E. S. et al. BRCA1 deficient mouse models to study pathogenesis and therapy of triple negative breast cancer. Breast Dis. 32, 85–97 (2010).
Duarte, A. A. et al. BRCA-deficient mouse mammary tumor organoids to study cancer-drug resistance. Nat. Methods 15, 134–140 (2018).
Sun, K. et al. A comparative pharmacokinetic study of PARP inhibitors demonstrates favorable properties for niraparib efficacy in preclinical tumor models. Oncotarget 9, 37080–37096 (2018).
Rao, T. et al. Inducible and coupled expression of the polyomavirus middle T antigen and Cre recombinase in transgenic mice: an in vivo model for synthetic viability in mammary tumour progression. Breast Cancer Res. 16, R11 (2014).
Hutter, C. & Zenklusen, J. C. The Cancer Genome Atlas: creating lasting value beyond its data. Cell 173, 283–285 (2018).
Yeo, S. K. et al. Single-cell RNA-sequencing reveals distinct patterns of cell state heterogeneity in mouse models of breast cancer. eLife 9, e58810 (2020).
Li, W., Germain, R. N. & Gerner, M. Y. High-dimensional cell-level analysis of tissues with Ce3D multiplex volume imaging. Nat. Protoc. 14, 1708–1733 (2019).
Olive, P. L. & Banath, J. P. The comet assay: a method to measure DNA damage in individual cells. Nat. Protoc. 1, 23–29 (2006).
Baslan, T. & Hicks, J. Unravelling biology and shifting paradigms in cancer with single-cell sequencing. Nat. Rev. Cancer 17, 557–569 (2017).
Dean, F. B. et al. Comprehensive human genome amplification using multiple displacement amplification. Proc. Natl Acad. Sci. USA 99, 5261–5266 (2002).
