Recent Advances in Organ-Targeting Polymeric Delivery Vectors for Nucleic Acids★
Received date: 2023-05-24
Online published: 2023-07-07
Supported by
National Science Fund for Distinguished Young Scholars, NSFC(21925505); Shanghai International Scientific Collaboration Fund(21520710100); Innovation Program of Shanghai Municipal Education Commission(2023ZKZD28); National Natural Science Foundation of China(51903190); National Natural Science Foundation of China(22175131)
Nucleic acids-based therapy has been developed rapidly in recent years and the effective treatment is closely related to the safe and efficient delivery of nucleic acids. On the one hand, it is difficult for nucleic acids to penetrate cell membranes and express. On the other hand, unlike other drugs, some genetic therapeutics work at the condition of expressing in specific organ or cells. Therefore, delivery vectors are particularly important: they are expected to protect nucleic acids and achieve efficient transfection. Besides, they should be able to target specific sites to increase the probability of nucleic acid expression in target cells. Given the importance of polymer nucleic acid vectors, in this review, the research progress of polymeric delivery systems is summarized, including how to achieve aggregation from the administration site to the target site, and the state of the art of organ-targeting polymer nucleic acid vectors. Finally, the perspective of organ-targeting polymeric nucleic acid vectors is given.
Xu Pu , Zejuan Li , Junqiu Shi , Yunqing Zhu , Jianzhong Du . Recent Advances in Organ-Targeting Polymeric Delivery Vectors for Nucleic Acids★[J]. Acta Chimica Sinica, 2023 , 81(10) : 1438 -1446 . DOI: 10.6023/A23050246
| [1] | van den Berg, A. I. S.; Yun, C. O.; Schiffelers, R. M.; Hennink, W. E. J. Controlled Release 2021, 331, 121. |
| [2] | Kulkarni, J. A.; Witzigmann, D.; Thomson, S. B.; Chen, S.; Leavitt, B. R.; Cullis, P. R.; van der Meel, R. Nat. Nanotechnol. 2021, 16, 630. |
| [3] | Guo, Z. P.; Lin, L.; Chen, J.; Tian, H. Y.; Chen, X. S. Chem. J. Chinese U. 2020, 41, 235 (in Chinese). |
| [3] | (郭兆培, 林琳, 陈杰, 田华雨, 陈学思, 高等学校化学学报, 2020, 41, 235.) |
| [4] | Kis, Z.; Kontoravdi, C.; Dey, A. K.; Shattock, R.; Shah, N. J. Adv. Manuf. Process. 2020, 2, e10060. |
| [5] | Weng, Y. H.; Li, C. H.; Yang, T. R.; Hu, B.; Zhang, M. J.; Guo, S.; Xiao, H. H.; Liang, X. J.; Huang, Y. Y. Biotechnol. Adv. 2020, 40, 107534. |
| [6] | Hajj, K. A.; Whitehead, K. A. Nat. Rev. Mater. 2017, 2, 17056. |
| [7] | Kowalski, P. S.; Rudra, A.; Miao, L.; Anderson, D. G. Mol. Ther. 2019, 27, 710. |
| [8] | Meyer, R. A.; Neshat, S. Y.; Green, J. J.; Santos, J. L.; Tuesca, A. D. Mater. Today Adv. 2022, 14, 100240. |
| [9] | Servick, K. Science 2020, 370, 1388. |
| [10] | Blakney, A. K.; Zhu, Y.; McKay, P. F.; Bouton, C. R.; Yeow, J.; Tang, J.; Hu, K.; Samnuan, K.; Grigsby, C. L.; Shattock, R. J.; Stevens, M. M. ACS Nano 2020, 14, 5711. |
| [11] | Han, X.; Zhang, L. W.; Zhang, Q.; Sui, X. H.; Qian, M.; Chen, Q. X.; Wang, J. Y. Acta Chim. Sinica 2021, 79, 794 (in Chinese). |
| [11] | (韩旭, 张留伟, 张强, 睢晞航, 钱明, 陈麒先, 王静云, 化学学报, 2021, 79, 794.) |
| [12] | Wang, H.; Chang, H.; Zhang, Q.; Cheng, Y. Top. Curr. Chem. 2017, 375, 62. |
| [13] | Kumar, R.; Santa Chalarca, C. F.; Bockman, M. R.; Van Bruggen, C.; Grimme, C. J.; Dalal, R. J.; Hanson, M. G.; Hexum, J. K.; Reineke, T. M. Chem. Rev. 2021, 121, 11527. |
| [14] | Mendes, B. B.; Conniot, J.; Avital, A.; Yao, D.; Jiang, X.; Zhou, X.; Sharf-Pauker, N.; Xiao, Y.; Adir, O.; Liang, H.; Shi, J.; Schroeder, A.; Conde, J. Nat. Rev. Methods Primers 2022, 2, 24. |
| [15] | Bus, T.; Traeger, A.; Schubert, U. S. J. Mater. Chem. B 2018, 6, 6904. |
| [16] | Pei, H.; Deng, H. Z.; Zhou, Y. F.; Chen, X. Y. Matter 2022, 5, 1670. |
| [17] | Park, T. G.; Jeong, J. H.; Kim, S. W. Adv. Drug Del. Rev. 2006, 58, 467. |
| [18] | Breunig, M.; Lungwitz, U.; Liebl, R.; Goepferich, A. Proc. Natl. Acad. Sci. U. S. A. 2007, 104, 14454. |
| [19] | Li, M.; Li, Y.; Peng, K.; Wang, Y.; Gong, T.; Zhang, Z. R.; He, Q.; Sun, X. Acta Biomater. 2017, 64, 237. |
| [20] | Green, J. J.; Langer, R.; Anderson, D. G. Acc. Chem. Res. 2008, 41, 749. |
| [21] | Chaudhary, N.; Weissman, D.; Whitehead, K. A. Nat. Rev. Drug Discovery 2021, 20, 817. |
| [22] | Kheraldine, H.; Rachid, O.; Habib, A. M.; Al Moustafa, A.-E.; Benter, I. F.; Akhtar, S. Adv. Drug Del. Rev. 2021, 178, 113908. |
| [23] | Laechelt, U.; Wagner, E. Chem. Rev. 2015, 115, 11043. |
| [24] | Cedervall, T.; Lynch, I.; Foy, M.; Berggard, T.; Donnelly, S. C.; Cagney, G.; Linse, S.; Dawson, K. A. Angew. Chem., Int. Ed. 2007, 46, 5754. |
| [25] | Lima, T.; Bernfur, K.; Vilanova, M.; Cedervall, T. Sci. Rep. 2020, 10, 1129. |
| [26] | Francia, V.; Schiffelers, R. M.; Cullis, P. R.; Witzigmann, D. Bioconjugate Chem. 2020, 31, 2046. |
| [27] | Mohammad-Beigi, H.; Hayashi, Y.; Zeuthen, C. M.; Eskandari, H.; Scavenius, C.; Juul-Madsen, K.; Vorup-Jensen, T.; Enghild, J. J.; Sutherland, D. S. Nat. Commun. 2020, 11, 4535. |
| [28] | Owens, D. E.,3rd; Peppas, N. A. Int. J. Pharm. 2006, 307, 93. |
| [29] | Akinc, A.; Querbes, W.; De, S.; Qin, J.; Frank-Kamenetsky, M.; Jayaprakash, K. N.; Jayaraman, M.; Rajeev, K. G.; Cantley, W. L.; Dorkin, J. R.; Butler, J. S.; Qin, L.; Racie, T.; Sprague, A.; Fava, E.; Zeigerer, A.; Hope, M. J.; Zerial, M.; Sah, D. W. Y.; Fitzgerald, K.; Tracy, M. A.; Manoharan, M.; Koteliansky, V.; de Fougerolles, A.; Maier, M. A. Mol. Ther. 2010, 18, 1357. |
| [30] | Dilliard, S. A.; Cheng, Q.; Siegwart, D. J. Proc. Natl. Acad. Sci. U. S. A. 2021, 118, e2109256118. |
| [31] | Song, T.; Xia, Y.; Du, Y.; Chen, M. W.; Qing, H.; Ma, G. Adv. Mater. 2021, 33, 2100106. |
| [32] | Cao, Y.; He, Z.; Chen, Q.; He, X.; Su, L.; Yu, W.; Zhang, M.; Yang, H.; Huang, X.; Li, J. Nano Lett. 2022, 22, 6580. |
| [33] | Blanco, E.; Shen, H.; Ferrari, M. Nat. Biotechnol. 2015, 33, 941. |
| [34] | Fang, C.; Shi, B.; Pei, Y. Y.; Hong, M. H.; Wu, J.; Chen, H. Z. Eur. J. Pharm. Sci. 2006, 27, 27. |
| [35] | Vegh, P.; Fletcher, J.; Dixon, D.; Haniffa, M., In Encyclopedia of Life Sciences, John Wiley and Sons, Hoboken, 2017, pp. 1-8. |
| [36] | Besin, G.; Milton, J.; Sabnis, S.; Howell, R.; Mihai, C.; Burke, K.; Benenato, K. E.; Stanton, M.; Smith, P.; Senn, J.; Hoge, S. ImmunoHorizons 2019, 3, 282. |
| [37] | Choi, H. S.; Liu, W.; Misra, P.; Tanaka, E.; Zimmer, J. P.; Ipe, B. I.; Bawendi, M. G.; Frangioni, J. V. Nat. Biotechnol. 2007, 25, 1165. |
| [38] | Poon, W.; Zhang, Y. N.; Ouyang, B.; Kingston, B. R.; Wu, J. L. Y.; Wilhelm, S.; Chan, W. C. W. ACS Nano 2019, 13, 5785. |
| [39] | Mitchell, M. J.; Billingsley, M. M.; Haley, R. M.; Wechsler, M. E.; Peppas, N. A.; Langer, R. Nat. Rev. Drug Discovery 2021, 20, 101. |
| [40] | Dilliard, S. A.; Siegwart, D. J. Nat. Rev. Mater. 2023, 8, 282. |
| [41] | Poisson, J.; Lemoinne, S.; Boulanger, C.; Durand, F.; Moreau, R.; Valla, D.; Rautou, P.-E. J. Hepatol. 2017, 66, 212. |
| [42] | Sato, Y.; Hatakeyama, H.; Hyodo, M.; Harashima, H. Mol. Ther. 2016, 24, 788. |
| [43] | Schudel, A.; Francis, D. M.; Thomas, S. N. Nat. Rev. Mater. 2019, 4, 415. |
| [44] | Trevaskis, N. L.; Kaminskas, L. M.; Porter, C. J. H. Nat. Rev. Drug Discovery 2015, 14, 781. |
| [45] | Hassett, K. J.; Higgins, J.; Woods, A.; Levy, B.; Xia, Y.; Hsiao, C. J.; Acosta, E.; Almarsson, O.; Moore, M. J.; Brito, L. A. J. Controlled Release 2021, 335, 237. |
| [46] | Kranz, L. M.; Diken, M.; Haas, H.; Kreiter, S.; Loquai, C.; Reuter, K. C.; Meng, M.; Fritz, D.; Vascotto, F.; Hefesha, H.; Grunwitz, C.; Vormehr, M.; Husemann, Y.; Selmi, A.; Kuhn, A. N.; Buck, J.; Derhovanessian, E.; Rae, R.; Attig, S.; Diekmann, J.; Jabulowsky, R. A.; Heesch, S.; Hassel, J.; Langguth, P.; Grabbe, S.; Huber, C.; Tureci, O.; Sahin, U. Nature 2016, 534, 396. |
| [47] | Zhao, J. Y.; Song, W. T.; Tang, Z. H.; Chen, X. S. Acta Chim. Sinica 2022, 80, 563 (in Chinese). |
| [47] | (赵佳雨, 宋万通, 汤朝晖, 陈学思, 化学学报, 2022, 80, 563.) |
| [48] | Li, X.; Guo, X.; Hu, M.; Cai, R.; Chen, C. J. Mater. Chem. B 2023, 11, 2063. |
| [49] | Mi, P.; Cabral, H.; Kataoka, K. Adv. Mater. 2020, 32, 1902604. |
| [50] | Li, J. J.; Kataoka, K. J. Am. Chem. Soc. 2021, 143, 538. |
| [51] | Fornaguera, C.; Guerra-Rebollo, M.; Angel Lazaro, M.; Castells- Sala, C.; Meca-Cortes, O.; Ramos-Perez, V.; Cascante, A.; Rubio, N.; Blanco, J.; Borros, S. Adv. Healthcare Mater. 2018, 7, 1800335. |
| [52] | Kaczmarek, J. C.; Patel, A. K.; Kauffman, K. J.; Fenton, O. S.; Webber, M. J.; Heartlein, M. W.; DeRosa, F.; Anderson, D. G. Angew. Chem. Int. Ed. 2016, 55, 13808. |
| [53] | Ke, X.; Shelton, L.; Hu, Y.; Zhu, Y.; Chow, E.; Tang, H.; Santos, J. L.; Mao, H. Q. ACS Appl. Mater. Interfaces 2020, 12, 35835. |
| [54] | Patel, A. K.; Kaczmarek, J. C.; Bose, S.; Kauffman, K. J.; Mir, F.; Heartlein, M. W.; DeRosa, F.; Langer, R.; Anderson, D. G. Adv. Mater. 2019, 31, 1805116. |
| [55] | Gilleron, J.; Querbes, W.; Zeigerer, A.; Borodovsky, A.; Marsico, G.; Schubert, U.; Manygoats, K.; Seifert, S.; Andree, C.; Stoter, M.; Epstein-Barash, H.; Zhang, L. G.; Koteliansky, V.; Fitzgerald, K.; Fava, E.; Bickle, M.; Kalaidzidis, Y.; Akinc, A.; Maier, M.; Zerial, M. Nat. Biotechnol. 2013, 31, 638. |
| [56] | Nicoli, E.; Syga, M. I.; Bosetti, M.; Shastri, V. P. PLoS One 2015, 10, e0122581. |
| [57] | Kermaniyan, S. S.; Chen, M.; Zhang, C.; Smith, S. A.; Johnston, A. P. R.; Such, C.; Such, G. K. Macromol. Biosci. 2022, 22, 2100445. |
| [58] | Trefts, E.; Gannon, M.; Wasserman, D. H. Curr. Biol. 2017, 27, R1147. |
| [59] | Rohner, E.; Yang, R.; Foo, K. S.; Goedel, A.; Chien, K. R. Nat. Biotechnol. 2022, 40, 1586. |
| [60] | Kristen, A. V.; Ajroud-Driss, S.; Conceicao, I.; Gorevic, P.; Kyriakides, T.; Obici, L. Neurodegener. Dis. Manag. 2019, 9, 5. |
| [61] | Lam, J. K.-W.; Liang, W.; Chan, H.-K. Adv. Drug Del. Rev. 2012, 64, 1. |
| [62] | Miao, L.; Zhang, Y.; Huang, L. Mol. Cancer 2021, 20, 41. |
| [63] | Densmore, C. L.; Orson, F. M.; Xu, B.; Kinsey, B. M.; Waldrep, J. C.; Hua, P.; Bhogal, B.; Knight, V. Mol. Ther. 2000, 1, 180. |
| [64] | Rudolph, C.; Lausier, J.; Naundorf, S.; Muller, R. H.; Rosenecker, J. J. Gene Med. 2000, 2, 269. |
| [65] | Suk, J. S.; Kim, A. J.; Trehan, K.; Schneider, C. S.; Cebotaru, L.; Woodward, O. M.; Boylan, N. J.; Boyle, M. P.; Lai, S. K.; Guggino, W. B.; Hanes, J. J. Controlled Release 2014, 178, 8. |
| [66] | Mastorakos, P.; da Silva, A. L.; Chisholm, J.; Song, E.; Choi, W. K.; Boyle, M. P.; Morales, M. M.; Hanes, J.; Suk, J. S. Proc. Natl. Acad. Sci. U. S. A. 2015, 112, 8720. |
| [67] | Elfinger, M.; Maucksch, C.; Rudolph, C. Biomaterials 2007, 28, 3448. |
| [68] | Rosenblum, D.; Joshi, N.; Tao, W.; Karp, J. M.; Peer, D. Nat. Commun. 2018, 9, 1410. |
| [69] | Chen, Y.; De Koker, S.; De Geest, B. G. Acc. Chem. Res. 2020, 53, 2055. |
| [70] | Jiang, Y. H.; Lu, Q.; Wang, Y. H.; Xu, E.; Ho, A.; Singh, P.; Wang, Y. F.; Jiang, Z. Z.; Yang, F.; Tietjen, G. T.; Cresswell, P.; Saltzman, W. M. Nano Lett. 2020, 20, 1117. |
| [71] | Grun, M. K.; Suberi, A.; Shin, K.; Lee, T.; Gomerdinger, V.; Moscato, Z. M.; Piotrowski-Daspit, A. S.; Saltzman, W. M. Biomaterials 2021, 272, 120780. |
| [72] | Huang, P.; Jiang, L. S.; Pan, H.; Ding, L. W.; Zhou, B.; Zhao, M. Y.; Zou, J. H.; Li, B. H.; Qi, M. W.; Deng, H. Z.; Zhou, Y. F.; Chen, X. Y. Adv. Mater. 2023, 35, 2207471. |
| [73] | Liu, S.; Wang, X.; Yu, X. L.; Cheng, Q.; Johnson, L. T.; Chatterjee, S.; Zhang, D.; Lee, S. M.; Sun, Y. H.; Lin, T. C.; Liu, J. L.; Siegwart, D. J. J. Am. Chem. Soc. 2021, 143, 21321. |
| [74] | Liu, S.; Cheng, Q.; Wei, T.; Yu, X. L.; Johnson, L. T.; Farbiak, L.; Siegwart, D. J. Nat. Mater. 2021, 20, 701. |
| [75] | Yang, R.; Deng, Y.; Huang, B.; Huang, L.; Lin, A.; Li, Y.; Wang, W.; Liu, J.; Lu, S.; Zhan, Z.; Wang, Y.; Ruhan, A.; Wang, W.; Niu, P.; Zhao, L.; Li, S.; Ma, X.; Zhang, L.; Zhang, Y.; Yao, W.; Liang, X.; Zhao, J.; Liu, Z.; Peng, X.; Li, H.; Tan, W. Signal Transduction Targeted Ther. 2021, 6, 213. |
| [76] | Segovia, N.; Dosta, P.; Cascante, A.; Ramos, V.; Borros, S. Acta Biomater. 2014, 10, 2147. |
| [77] | Smith, T. T.; Stephan, S. B.; Moffett, H. F.; McKnight, L. E.; Ji, W. H.; Reiman, D.; Bonagofski, E.; Wohlfahrt, M. E.; Pillai, S. P. S.; Stephan, M. T. Nat. Nanotechnol. 2017, 12, 813. |
| [78] | Parayath, N. N.; Stephan, S. B.; Koehne, A. L.; Nelson, P. S.; Stephan, M. T. Nat. Commun. 2020, 11, 6080. |
| [79] | Kim, H. J.; Seo, S. K.; Park, H. Y. J. Controlled Release 2022, 345, 405. |
/
| 〈 |
|
〉 |
