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Laboratory of Gene Therapy
Prof. dr. Niek Sanders is the head of the Laboratory of Gene Therapy (LGT, Ghent University). His team currently consisted out of 12 researchers and one administrative manager. They mainly focus on the preclinical investigation of mRNA therapeutics and mRNA vaccines. With regard to the design of mRNA therapeutics, Sanders’s team is one of the leading teams in the world. They use several synthetic mRNA platforms (unmodified, chemical modified and self-replication mRNAs) and together with MIT they engineered self-replicating mRNAs that can be switched ON and OFF on-demand in vitro as well as in vivo. Sanders’s team was the first to publish a paper reporting in detail on the beneficial effects of N(1)-methylpseudouridine modified mRNA. This modification is used in the mRNA-based SARS-CoV-2 vaccines and it is crucial for their activity. The team also has a strong focus on the investigation of novel polymer and lipid nanoparticle carriers for mRNA vaccines. The diseases that they target are mainly cancer and infections. To that end murine models for melanoma, triple negative breast cancer, lung cancer and colon cancer are used. Moreover, the team has also access to large animal cancer models such dogs and cats with spontaneous cancer. For the evaluation of their mRNA vaccine leads against pathogens they use murine models or the target species in case of veterinary vaccines. The readouts that the team routinely performs to measure the efficacy of the mRNA vaccines are: mRNA expression studies (in vitro & in vivo), biodistribution studies, and assays that measure the innate and adaptive immune responses elicited by mRNA vaccines.