Associate Professor Lefkothea Papadopoulou

PI / Investigator: Associate Professor Lefkothea Papadopoulou - Aristotle University of Thessaloniki, Greece.

Award Type: Award for Innovative Mindset made possible through funding by fara New Zealand.

Grant Title: Delivery of Frataxin in vitro transcribed mRNAs as a therapeutic approach for Friedreich ataxia.

Lay Summary:

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 FRDA.

Here we propose to apply our innovative PTD-IVT-mRNA delivery platform to the treatment of FRDA. This is a fusion between the mRNA expressing frataxin and a small peptide that will facilitate the internalization of the FXN mRNA, resulting in its translation inside the cell and production of frataxin. We are going to test this technology in “mini organs” in a dish derived from human induced pluripotent stem cell from FRDA patients.