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

  • 1.

    Adams, D. et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N. Engl. J. Med. 379, 11–21 (2018).

    CAS 

    Google Scholar 

  • 2.

    Garrelfs, S. F. et al. Lumasiran, an RNAi therapeutic for primary hyperoxaluria type 1. N. Engl. J. Med. 384, 1216–1226 (2021).

    CAS 

    Google Scholar 

  • 3.

    Balwani, M. et al. Phase 3 trial of RNAi therapeutic givosiran for acute intermittent porphyria. N. Engl. J. Med. 382, 2289–2301 (2020).

    CAS 

    Google Scholar 

  • 4.

    Ray, K. K. et al. Two phase 3 trials of inclisiran in patients with elevated LDL cholesterol. N. Engl. J. Med. 382, 1507–1519 (2020).

    CAS 

    Google Scholar 

  • 5.

    Finkel, R. S. et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N. Engl. J. Med. 377, 1723–1732 (2017).

    CAS 

    Google Scholar 

  • 6.

    Baden, L. R. et al. Efficacy and safety of the mRNA-1273 SARS-CoV-2 vaccine. N. Engl. J. Med. 384, 403–416 (2020).

    Google Scholar 

  • 7.

    Polack, F. P. et al. Safety and efficacy of the BNT162b2 mRNA Covid-19 vaccine. N. Engl. J. Med. 383, 2603–2615 (2020).

    CAS 

    Google Scholar 

  • 8.

    Sahin, U. et al. Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer. Nature 547, 222–226 (2017).

    CAS 

    Google Scholar 

  • 9.

    Nair, J. K. et al. Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J. Am. Chem. Soc. 136, 16958–16961 (2014).

    CAS 

    Google Scholar 

  • 10.

    Akinc, A. et al. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nat. Nanotechnol. 14, 1084–1087 (2019).

    CAS 

    Google Scholar 

  • 11.

    Akinc, A. et al. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol. Ther. 18, 1357–1364 (2010).

    CAS 

    Google Scholar 

  • 12.

    Willoughby, J. L. S. et al. Evaluation of GalNAc-siRNA conjugate activity in pre-clinical animal models with reduced asialoglycoprotein receptor expression. Mol. Ther. 26, 105–114 (2018).

    CAS 

    Google Scholar 

  • 13.

    Lisowski, L. et al. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 506, 382–386 (2014).

    CAS 

    Google Scholar 

  • 14.

    Paulk, N. K. et al. Bioengineered AAV capsids with combined high human liver transduction in vivo and unique humoral seroreactivity. Mol. Ther. 26, 289–303 (2018).

    CAS 

    Google Scholar 

  • 15.

    Vercauteren, K. et al. Superior in vivo transduction of human hepatocytes using engineered AAV3 capsid. Mol. Ther. 24, 1042–1049 (2016).

    CAS 

    Google Scholar 

  • 16.

    Pei, X. et al. Development of AAV variants with human hepatocyte tropism and neutralizing antibody escape capacity. Mol. Ther. Methods Clin. Dev. 18, 259–268 (2020).

    CAS 

    Google Scholar 

  • 17.

    Wilson, E. M. et al. Extensive double humanization of both liver and hematopoiesis in FRGN mice. Stem Cell Res. 13, 404–412 (2014).

    CAS 

    Google Scholar 

  • 18.

    Foquet, L. et al. Successful engraftment of human hepatocytes in uPA-SCID and FRG® KO mice. Methods Mol. Biol. 1506, 117–130 (2017).

    CAS 

    Google Scholar 

  • 19.

    Chen, D. et al. Rapid discovery of potent siRNA-containing lipid nanoparticles enabled by controlled microfluidic formulation. J. Am. Chem. Soc. 134, 6948–6951 (2012).

    CAS 

    Google Scholar 

  • 20.

    Sago, C .D. et al. Modifying a commonly expressed endocytic receptor retargets nanoparticles in vivo. Nano Lett. 18, 7590–7600 (2018).

    CAS 

    Google Scholar 

  • 21.

    Sago, C. D. et al. Nanoparticles that deliver RNA to bone marrow identified by in vivo directed evolution. J. Am. Chem. Soc. 140, 17095–17105 (2018).

    CAS 

    Google Scholar 

  • 22.

    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).

    CAS 

    Google Scholar 

  • 23.

    Tiwari, P. M. et al. Engineered mRNA-expressed antibodies prevent respiratory syncytial virus infection. Nat. Commun. 9, 3999 (2018).

    Google Scholar 

  • 24.

    Paunovska, K. et al. Nanoparticles containing oxidized cholesterol deliver mRNA to the liver microenvironment at clinically relevant doses. Adv. Mater. 31, e1807748 (2019).

    Google Scholar 

  • 25.

    Dong, Y. et al. Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates. Proc. Natl Acad. Sci. USA 111, 3955–3960 (2014).

    CAS 

    Google Scholar 

  • 26.

    Dahlman, J. E. et al. In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight. Nat. Nanotechnol. 9, 648–655 (2014).

    CAS 

    Google Scholar 

  • 27.

    Lokugamage, M. P. et al. Mild innate immune activation overrides efficient nanoparticle-mediated RNA delivery. Adv. Mater. 32, 1904905 (2020).

    CAS 

    Google Scholar 

  • 28.

    Paunovska, K. et al. Analyzing 2000 in vivo drug delivery data points reveals cholesterol structure impacts nanoparticle delivery. ACS Nano 12, 8341–8349 (2018).

    CAS 

    Google Scholar 

  • 29.

    Patel, S. et al. Naturally-occurring cholesterol analogues in lipid nanoparticles induce polymorphic shape and enhance intracellular delivery of mRNA. Nat. Commun. 11, 983 (2020).

    CAS 

    Google Scholar 

  • 30.

    Mui, B. L. et al. Influence of polyethylene glycol lipid desorption rates on pharmacokinetics and pharmacodynamics of siRNA lipid nanoparticles. Mol. Ther. Nucleic Acids 2, e139 (2013).

    CAS 

    Google Scholar 

  • 31.

    Kaczmarek, J. C. et al. Optimization of a degradable polymer-lipid nanoparticle for potent systemic delivery of mRNA to the lung endothelium and immune cells. Nano Lett. 18, 6449–6454 (2018).

    CAS 

    Google Scholar 

  • 32.

    Kranz, L. M. et al. Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy. Nature 534, 396–401 (2016).

    Google Scholar 

  • 33.

    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).

    CAS 

    Google Scholar 

  • 34.

    Lokugamage, M. P., Sago, C. D. & Dahlman, J. E. Testing thousands of nanoparticles in vivo using DNA barcodes. Curr. Opin. Biomed. Eng. 7, 1–8 (2018).

    Google Scholar 

  • 35.

    Patel, S. et al. Boosting intracellular delivery of lipid nanoparticle-encapsulated mRNA. Nano Lett. 17, 5711–5718 (2017).

    CAS 

    Google Scholar 

  • 36.

    Picelli, S. et al. Full-length RNA-seq from single cells using Smart-seq2. Nat. Protoc. 9, 171–181 (2014).

    CAS 

    Google Scholar 

  • 37.

    Ge, S. X., Son, E. W. & Yao, R. iDEP: an integrated web application for differential expression and pathway analysis of RNA-Seq data. BMC Bioinformatics 19, 534 (2018).

    CAS 

    Google Scholar 

  • 38.

    Low, J. Z. B., Khang, T. F. & Tammi, M. T. CORNAS: coverage-dependent RNA-Seq analysis of gene expression data without biological replicates. BMC Bioinformatics 18, 575 (2017).

    Google Scholar 

  • 39.

    Huang, D. W., Sherman, B. T. & Lempicki, R. A. Systematic and integrative analysis of large gene lists using DAVID bioinformatics resources. Nat. Protoc. 4, 44–57 (2009).

    CAS 

    Google Scholar 

  • 40.

    Szklarczyk, D. et al. The STRING database in 2017: quality-controlled protein–protein association networks, made broadly accessible. Nucleic Acids Res. 45, D362–D368 (2017).

    CAS 

    Google Scholar 

  • 41.

    Dobrovolskaia, M. A., Shurin, M. & Shvedova, A. A. Current understanding of interactions between nanoparticles and the immune system. Toxicol. Appl. Pharmacol. 299, 78–89 (2016).

    CAS 

    Google Scholar 

  • 42.

    Azuma, H. et al. Robust expansion of human hepatocytes in Fah–/–/Rag2–/–/Il2rg–/– mice. Nat. Biotechnol. 25, 903–910 (2007).

    CAS 

    Google Scholar 

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