Date: 24th March 2026
Time: Time: 12.30 – 1.30
Speaker: Dr Sonam Gurung. Postdoctoral Researcher at University College London (UCL)
Title: “messenger RNA (mRNA) therapy in inherited metabolic disorders”
Abstract:
My research is focussed on investigating novel therapies in urea cycle defects (UCD) and neurometabolic disorders, and improving the understanding of mechanisms underlying disease pathology. I am particularly focussed on investigating therapeutic application of mRNA therapies encapsulated in lipid nanoparticles (LNPs). mRNA-LNPs are leading non-viral gene therapy strategy, rapidly expanding for translation in vaccines and therapeutics, especially for rare genetic diseases. mRNA therapy can provide a temporary therapeutic effect with the flexibility to tailor treatment according to clinical requirements. mRNA-LNPs platform has been implemented successfully in multiple preclinical programs, with application ranging from protein replacement to nuclease-mediated gene silencing, editing or integration strategies. I will aim to review the rapid expansion of RNA biotherapeutics, the basics of LNP-mRNA technology, its advantages and limitations compared to viral gene therapy, with a spotlight on our preclinical work in argininosuccinic aciduria.
