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Lipid nanoparticle (LNP) delivery of gene therapy in inflammatory disease

Start year: 2024

Summary: The broader project, led by WEHI, will use lipid nanoparticle (LNP) technology to deliver immunosuppressive gene therapy in proof-of-concept studies for autoimmune and inflammatory disease. We propose to increase intracellular SOCS protein levels, using an LNP-mRNA delivery system, to switch off damaging inflammatory signalling. This will be evaluated in relevant cell lines and focused disease models, including suitable alternatives to mouse models of COPD, TEN and SLE. If successful, our approach of LNP-delivered Socs1 mRNA could be applied to many inflammatory and autoimmune diseases, where there is an ongoing unmet need for fine-tuned therapies with reduced side-effects. The UTS component will deliver on Aim 2 - focusing on the inflammatory lung disease models. Suitable models will be discussed and agreed upon with the lead researcher at WEHI upon confirmation of the funding, to replace the previously used mouse models.