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Development of novel ionizable lipids for lipid nanoparticle-mediated gene delivery
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School of Chemical Engineering |
Master's thesis
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en
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46
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Abstract
pDNA-based gene delivery using lipid nanoparticles (LNPs) holds significant promise for long-lasting treatments of a broad range of diseases, including cancer and diabetes, through single-dose or infrequent administration – unlike RNA-based therapies that typi-cally require frequent or repeated dosing. While several LNP formulations have already been approved for therapeutic use of RNA delivery (e.g. mRNA COVID-19 vaccines), DNA delivery presents additional challenges given the larger size and distinct nature of the genetic cargo. As the main components of LNPs, ionizable lipids play a crucial role in ensuring targeted delivery of therapeutic material to specific tissues. Developing function-al ionizable lipids is essential for the safe and effective transport and release of nucleic acid. In the context of liver-targeted gene delivery for treating Citrullinemia Type I, we present novel lipopeptides featuring ionizable sites at the C-terminus of their head as well as branched lipidic tails. This architecture possesses promising capabilities in protecting DNA cargo and enabling efficient release within liver hepatocytes.