Polymers for RNA delivery Valentin Fell and Olivia Merkel LMU Munich, Germany
RNA therapy offer new approaches to treat diseases deemed as “undruggable” in the past. As famous example, the mRNA-based COVID-19 vaccines have played a prominent role in the mitigation of the COVID-19 pandemic. Also, other diseases like rheumatic diseases, 1 cancer, 2 and asthma 3 might be able to be targeted in the future. Basically, with mRNA, a cell can be brought to produce a specific protein, 4 whilst the introduction of siRNA can help to silence a gene leading to e.g. asthma. 3 However, the intrinsic problem with RNA therapy is the low stability of RNA in the body; the omnipresent RNases disassemble RNA strings. 5 Moreover, the negatively charged RNA strings would be repelled by the also negatively charged cell membrane. 2 As such, carrier systems are required which encapsulate the RNA strings, which protect the RNA from decomposition and support the entry in the cell, foremostly by the formation of an endosome. Inside the cell, the carrier system should support the escape of the particle out of the endosome and should release the RNA. 3 We in the research group of Prof. Olivia Merkel work on the synthesis of novel carrier systems for RNA delivery. The focus lies on polymers, e.g. poly(β-amino esters). 6 The poster to be presented on the conference will give an overview of the overall research topic as described and will give examples for polymers applied, which are also subject of research in the Lab of Prof. Merkel. References 1. S. K. Jones, V. Lizzio and O. M. Merkel, Biomacromolecules , 2016, 17 , 76-87. 2. Y. Xie, N. H. Kim, V. Nadithe, D. Schalk, A. Thakur, A. Kılıç, L. G. Lum, D. J. P. Bassett and O. M. Merkel, J. Controlled Release , 2016, 229 , 120-129. 3. S. Qin, X. Tang, Y. Chen, K. Chen, N. Fan, W. Xiao, Q. Zheng, G. Li, Y. Teng, M. Wu and X. Song, Signal Transduction and Targeted Therapy , 2022, 7 , 166. 4. C. E. Pugsley, R. E. Isaac, N. J. Warren, J. S. Behra, K. Cappelle, R. Dominguez-Espinosa and O. J. Cayre, Biomacromolecules , 2022, 23 , 2362-2373. 5. K. L. Kozielski, S. Y. Tzeng and J. J. Green, Chem. Commun. , 2013, 49 , 5319-5321.
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