Extracellular Vesicles - Roles in mRNA-Based Therapies

dc.contributor.authorTangruksa, Benyapa
dc.date.accessioned2025-05-20T07:25:48Z
dc.date.available2025-05-20T07:25:48Z
dc.date.issued2025-05-20
dc.description.abstractEfficient and targeted delivery of therapeutic mRNA remains a major challenge in RNA-based medicine. This thesis explores how lipid nanoparticles (LNPs) and extracellular vesicles (EVs) cooperate in the delivery and redistribution of vascular endothelial growth factor A (VEGFA) mRNA, therapeutic mRNA that induces blood vessel formation and holds strong potential for treating cardiovascular diseases through regenerative angiogenesis. Paper I demonstrates that LNPs efficiently deliver VEGFA mRNA to human cells, leading to rapid uptake and translation. A portion of the internalized mRNA is subsequently secreted within EVs, which also acquire enriched proangiogenic transcripts. Among tested EVs, those derived from cardiac progenitor cells (CPC-EVs) showed superior angiogenic potential and minimal inflammatory response. In Paper II, biodistribution studies in mice revealed that CPC-EVs outperformed LNPs and non-cardiac EVs in delivering VEGFA mRNA to heart tissue, with reduced liver accumulation. CPC-EVs induced local protein expression and minimal transcriptomic disruption, highlighting their tissue-targeting advantage and safety. Paper III investigates how LNP treatment alters EV transcriptomes and how these modified EVs affect recipient tissues. VEGFA mRNA was the most enriched transcript across all EV types. CPC-EVs upregulated VEGFR-1 and VEGFR-2 specifically in cardiac tissue. Further analysis revealed that human mRNA transcripts were detectable in recipient mouse hearts. In summary, this thesis explores the interplay between LNPs and EVs in transporting VEGFA mRNA for therapeutic angiogenesis. LNPs enable efficient cellular uptake and subsequently trigger re-packaging of mRNA into EVs. CPC-EVs showed superior heart targeting and angiogenic potential with low inflammation. The findings highlight a promising strategy for regenerative treatment of cardiovascular diseases.sv
dc.gup.defencedate2025-06-12
dc.gup.defenceplaceTorsdagen den 12 juni 2025, kl. 13.00, Föreläsningssalen våning 3, Guldhedsgatan 10A, Göteborgsv
dc.gup.departmentInstitute of Medicine. Department of Rheumatology and Inflammation Researchsv
dc.gup.dissdb-fakultetSA
dc.gup.originUniversity of Gothenburg. Sahlgrenska Academysv
dc.identifier.isbn978-91-8115-262-3 (PRINT)
dc.identifier.isbn978-91-8115-263-0 (PDF)
dc.identifier.urihttps://hdl.handle.net/2077/85349
dc.language.isoengsv
dc.relation.haspartI. Lipid nanoparticles deliver the therapeutic VEGFA mRNA in vitro and in vivo and transform extracellular vesicles for their functional extensions. Nawaz, M., Heydarkhan‐Hagvall, S., Tangruksa, B., González‐King Garibotti, H., Jing, Y., Maugeri, M., Kohl, F., Hultin, L., Reyahi, A., Camponeschi, A., Kull, B., Christoffersson, J., Grimsholm, O., Jennbacken, K., Sundqvist, M., Wiseman, J., Bidar, A. W., Lindfors, L., Synnergren, J., & Valadi, H. Advanced Science. 2023 Apr;10(12):2206187. https://doi.org/10.1002/advs.202206187sv
dc.relation.haspartII. Targeted delivery of mRNA to the heart via extracellular vesicles or lipid nanoparticles. Nawaz, M., Tangruksa, B., Heydarkhan‐Hagvall, S., Kohl, F., González‐King Garibotti, H., Jing, Y., Payandeh, Z., Reyahi, A., Jennbacken, K., Wiseman, J., Hultin, L., Lindfors, L., Synnergren, J., & Valadi, H. BioRxiv. 2025:2025-01. https://doi.org/10.1101/2025.01.25.634881sv
dc.relation.haspartIII. Transcriptomic characterization of extracellular vesicles (EVs) and the tissue response following VEGFA mRNA delivery via EVs. Tangruksa, B., Nawaz, M., Heydarkhan‐Hagvall, S., Jin, T., Wiseman, J., Lindfors, L., Synnergren, J., & Valadi, H. Manuscriptsv
dc.subjectmRNA therapeuticssv
dc.subjectextracellular vesiclessv
dc.subjectlipid nanoparticlessv
dc.subjectexosomessv
dc.subjectVEGFAsv
dc.subjectVEGF receptorssv
dc.subjecttargeted deliverysv
dc.subjectangiogenesissv
dc.subjectcardiac progenitor cellssv
dc.subjecttranscriptomicssv
dc.subjectRNA sequencingsv
dc.subjectregenerative medicinesv
dc.titleExtracellular Vesicles - Roles in mRNA-Based Therapiessv
dc.typetexteng
dc.type.degreeDoctor of Philosophy (Medicine)sv
dc.type.svepDoctoral thesiseng

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