LAY SUMMARY

Can we use what we learned from COVID vaccines to develop a therapy for FA?

The 2023 Nobel Prize in Physiology or Medicine was awarded to Katalin Karikó and Drew Weissman for their groundbreaking findings concerning the nucleoside base modifications research that contributed to the development of effective mRNA vaccines against the SARS-COV-2 during the COVID-19 pandemic. More recently, the rise of mRNA technologies, have demonstrated conversion of human cells in protein factories, either via vaccination or through innovative mRNA therapeutics. Recombinant technologies have provided efficient Protein Replacement Therapies for several metabolic pathologies, such as diabetes mellitus. One of such approaches is being explored for FA. Here Dr Papadopoulou proposes to apply an innovative mRNA delivery platform to the treatment of FA. This is a conjugation between the mRNA expressing frataxin and a small peptide (PTD: Protein Transduction Domain) that will facilitate the internalization of the FXN mRNA, resulting in its translation inside the cell and subsequent production of frataxin. Dr Papadopoulou will test this technology in cell cultures and “mini organs” in a dish, derived from human induced pluripotent stem cell from FA patients.