Can we grow a brain in a dish to study FA?
Modelling Friedreich Ataxia in human iPSC-derived cerebellar organoid
Nerve cells in the cerebellum are particularly susceptible to damage in Friedreich's ataxia (FA). Understanding why these specific cells are vulnerable may help to develop treatments for FA that target these vulnerabilities.
The #FARAGrantProgram is pleased to award a grant to Dr. Esther Becker at the University of Oxford. Dr. Becker's lab will create 3D models of the human cerebellum in a dish. These ‘cerebellar organoids' are generated by first reprogramming cells obtained from a skin biopsy into Induced Pluripotent Stem Cells (IPSCs). IPSCs, which are capable of turning into any cell type in the body, are then turned into the cells of the cerebellum and grown to create a 3D model.
This project aims to extensively characterize the damage that occurs in the FA cerebellum using these cerebellar organoids. Dr. Becker's lab will also use this new understanding of the damage to test potential therapeutic compounds.
Thank you to the FA community for contributing to research like this by volunteering for skin biopsies!