Block catiomers with flanking hydrolyzable tyrosinate groups enhance in vivo mRNA delivery via π–π stacking-assisted micellar assembly

References

• Chabanovska O, Galow AM, David R, et al. mRNA - a game changer in regenerative medicine, cell-based therapy and reprogramming strategies. Adv Drug Deliv Rev. 2021 Dec;179:114002.
   PubMed Web of Science ®Google Scholar
• Lin CY, Perche F, Ikegami M, et al. Messenger RNA-based therapeutics for brain diseases: an animal study for augmenting clearance of beta-amyloid by intracerebral administration of neprilysin mRNA loaded in polyplex nanomicelles. J Control Release. 2016 Aug 10;235:268–13.
   PubMed Web of Science ®Google Scholar
• Sahin U, Kariko K, Tureci O. mRNA-based therapeutics–developing a new class of drugs. Nat Rev Drug Discov. 2014 Oct;13(10):759–780.
   PubMed Web of Science ®Google Scholar
• Polack FP, Thomas SJ, Kitchin N, et al. Safety and efficacy of the BNT162b2 mRNA Covid-19 vaccine. N Engl J Med. 2020 Dec 31;383(27):2603–2615.
   PubMed Web of Science ®Google Scholar
• Shroff RT, Chalasani P, Wei R, et al. Immune responses to two and three doses of the BNT162b2 mRNA vaccine in adults with solid tumors. Nat Med. 2021 Nov;27(11):2002–2011.
   PubMed Web of Science ®Google Scholar
• Tavernier G, Andries O, Demeester J, et al. mRNA as gene therapeutic: how to control protein expression. J Control Release. 2011 Mar 30;150(3):238–247.
   PubMed Web of Science ®Google Scholar
• Kowalski PS, Rudra A, Miao L, et al. Delivering the messenger: advances in technologies for therapeutic mRNA delivery. Mol Ther. 2019 Apr 10;27(4):710–728.
   PubMed Web of Science ®Google Scholar
• Uchida S, Perche F, Pichon C, et al. Nanomedicine-Based approaches for mRNA delivery. Mol Pharm. 2020 Oct 5;17(10):3654–3684.
   PubMed Web of Science ®Google Scholar
• Yang Y, Zhang M, Song H, et al. Silica-based nanoparticles for biomedical applications: from nanocarriers to biomodulators. Acc Chem Res. 2020 Aug 18;53(8):1545–1556.
   PubMed Web of Science ®Google Scholar
• Levacic AK, Berger S, Muller J, et al. Dynamic mRNA polyplexes benefit from bioreducible cleavage sites for in vitro and in vivo transfer. J Controlled Release. 2021 Nov 10;339:27–40.
   PubMed Web of Science ®Google Scholar
• Cheng Q, Wei T, Farbiak L, et al. Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-cas gene editing. Nat Nanotechnol. 2020 Apr;15(4):313–320.
   PubMed Web of Science ®Google Scholar
• Cabral H, Miyata K, Osada K, et al. Block copolymer micelles in nanomedicine applications. Chem Rev. 2018 Jul 25;118(14):6844–6892.
   PubMed Web of Science ®Google Scholar
• Jarak I, Pereira-Silva M, Santos AC, et al. Multifunctional polymeric micelle-based nucleic acid delivery: current advances and future perspectives. Appl Mater Today. 2021 Dec 25;25:101217.
   Web of Science ®Google Scholar
• Kartha G, Bello J, Harker D. Tertiary structure of ribonuclease. Nature. 1967 Mar 4;213(5079):862–865.
   PubMed Web of Science ®Google Scholar
• Burke RS, Pun SH. Extracellular barriers to in vivo PEI and PEGylated PEI polyplex-mediated gene delivery to the liver. Bioconjug Chem. 2008 Mar;19(3):693–704.
   PubMed Web of Science ®Google Scholar
• Zuckerman JE, Choi CH, Han H, et al. Polycation-siRNA nanoparticles can disassemble at the kidney glomerular basement membrane. Proc Natl Acad Sci USA. 2012 Feb 21;109(8):3137–3142.
   PubMed Web of Science ®Google Scholar
• Dirisala A, Uchida S, Tockary TA, et al. Precise tuning of disulphide crosslinking in mRNA polyplex micelles for optimising extracellular and intracellular nuclease tolerability. J Drug Target. 2019 Jun-Jul;27(5–6):670–680.
   PubMed Web of Science ®Google Scholar
• Yoshinaga N, Uchida S, Dirisala A, et al. mRNA loading into ATP-responsive polyplex micelles with optimal density of phenylboronate ester crosslinking to balance robustness in the biological milieu and intracellular translational efficiency. J Controlled Release. 2021 Feb 10;330:317–328.
   PubMed Web of Science ®Google Scholar
• Yoshinaga N, Uchida S, Dirisala A, et al. Bridging mRNA and polycation using mRNA Oligonucleotide derivatives improves the robustness of polyplex micelles for efficient mRNA delivery. Adv Healthc Mater. 2022 May;11(9):e2102016.
   PubMed Web of Science ®Google Scholar
• Yang W, Chen P, Boonstra E, et al. Polymeric micelles with pH-responsive cross-linked core enhance in vivo mRNA delivery. Pharmaceutics. 2022 Jun 6;14(6):1205.
   PubMed Web of Science ®Google Scholar
• Miyazaki T, Uchida S, Nagatoishi S, et al. Polymeric nanocarriers with controlled chain flexibility boost mRNA delivery in vivo through enhanced structural fastening. Adv Healthc Mater. 2020 Aug;9(16):e2000538.
   PubMed Web of Science ®Google Scholar
• Miyazaki T, Uchida S, Hatano H, et al. Guanidine-phosphate interactions stabilize polyion complex micelles based on flexible catiomers to improve mRNA delivery. Eur Polym J. 2020 Nov 5;140:110028.
   Web of Science ®Google Scholar
• Wilson KA, Holland DJ, Wetmore SD. Topology of RNA-protein nucleobase-amino acid pi-pi interactions and comparison to analogous DNA-protein pi-pi contacts. RNA. 2016 May;22(5):696–708.
   PubMed Web of Science ®Google Scholar
• Wilson KA, Kung RW, D’Souza S, et al. Anatomy of noncovalent interactions between the nucleobases or ribose and pi-containing amino acids in RNA-protein complexes. Nucleic Acids Res. 2021 Feb 26;49(4):2213–2225.
   PubMed Web of Science ®Google Scholar
• Kataoka K, Togawa H, Harada A, et al. Spontaneous formation of polyion complex micelles with narrow distribution from antisense oligonucleotide and cationic block copolymer in physiological saline. Macromolecules. 1996 Jan 1;29(26):8556–8557.
   Web of Science ®Google Scholar
• Thompson N. Topics in fluorescence spectroscopy. Techniques. 1991;1:337.
   Google Scholar
• Ruponen M, Yla-Herttuala S, Urtti A. Interactions of polymeric and liposomal gene delivery systems with extracellular glycosaminoglycans: physicochemical and transfection studies. Biochim Biophys Acta. 1999 Jan 8;1415(2):331–341.
   PubMed Web of Science ®Google Scholar
• Schmitt S, Nuhn L, Barz M, et al. Shining light on polymeric drug nanocarriers with fluorescence correlation spectroscopy. Macromol Rapid Commun. 2022 Feb;43(12):e2100892.
   PubMed Web of Science ®Google Scholar
• Rodriguez PC, Quiceno DG, Zabaleta J, et al. Arginase I production in the tumor microenvironment by mature myeloid cells inhibits T-cell receptor expression and antigen-specific T-cell responses. Cancer Res. 2004 Aug 15;64(16):5839–5849.
   PubMed Web of Science ®Google Scholar
• Zhou Z, Murdoch WJ, Shen Y. Synthesis of an esterase-sensitive degradable polyester as facile drug carrier for cancer therapy. J Polym Sci A Polym Chem. 2016;54(4):507–515.
   Web of Science ®Google Scholar
• Fernando IR, Ferris DP, Frasconi M, et al. Esterase- and pH-responsive poly(beta-amino ester)-capped mesoporous silica nanoparticles for drug delivery. Nanoscale. 2015 Apr 28;7(16):7178–7183.
   PubMed Web of Science ®Google Scholar
• Uchida S, Kinoh H, Ishii T, et al. Systemic delivery of messenger RNA for the treatment of pancreatic cancer using polyplex nanomicelles with a cholesterol moiety. Biomaterials. 2016 Mar;82:221–228.
   PubMed Web of Science ®Google Scholar
• Gan XJ, Liu SP, Liu ZF, et al. Fluorescence quenching method for the determination of carbazochrome sodium sulfonate with aromatic amino acids. Luminescence. 2013 May-Jun;28(3):265–269.
   PubMed Web of Science ®Google Scholar
• Gan X, Liu S, Liu Z, et al. Determination of tetracaine hydrochloride by fluorescence quenching method with some aromatic amino acids as probes. J Fluoresc. 2012 Jan;22(1):129–135.
   PubMed Web of Science ®Google Scholar
• Kauffman WB, Guha S, Wimley WC. Synthetic molecular evolution of hybrid cell penetrating peptides. Nat Commun. 2018 Jul 2;9(1):2568.
   PubMedGoogle Scholar
• Wang F, Hu K, Cheng Y. Structure-activity relationship of dendrimers engineered with twenty common amino acids in gene delivery. Acta Biomater. 2016 Jan;29:94–102.
   PubMed Web of Science ®Google Scholar
• Creusat G, Zuber G. Self-assembling polyethylenimine derivatives mediate efficient siRNA delivery in mammalian cells. Chembiochem. 2008 Nov 24;9(17):2787–2789.
   PubMed Web of Science ®Google Scholar