mRNA Therapeutics

mRNA therapeutics platform serves as a potential alternative to chemical drugs and gene therapy. We plan to focus on rare metabolic and genetic disorders which currently lack effective accessible therapies. mRNAs specifically targeting the disease of interest will be synthesised, assembled into Lipid nanoparticles (LNPs) purifies and tested for effective delivery in cell line as well as animal models. The assembled mRNA-LNPS will be characterized using Reversed-Phase High-Performance Liquid Chromatography (RP-HPLC). The mRNA and LNPs design and delivery will be optimized based on the disease and target tissue. The end-to-end capabilities include target specific mRNA design, synthesis, assembly into specific LNPs, use of receptors for tissue-specific targeted delivery of mRNA-LNPS as well as testing in cell lines and animal disease models.

Platform Coordinator: Bhagyashree Kaduskar

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