Can immune cells of the brain be used to rescue neurons in FA?
Dr. Stephanie Cherqui at the University of California, San Diego, has previously shown that gene editing of stem cells could be done outside of the body to repair the frataxin gene. Transplanting these edited cells into the body corrected some of the complications of Friedreich’s ataxia (FA) in a mouse model.
She hypothesized that these edited stem cells differentiated into brain immune cells (microglia) and that the improvement of the mice was due to both the repair of the microglia and the transfer of frataxin from the healthy microglia into diseased neurons.
We are pleased to award a grant to Dr. Cherqui to continue this work by investigating how microglia contribute to FA and whether gene editing of stem cells can help treat the disease.
This work will advance our understanding of microglia-neuron interactions and provide supporting evidence for the development of gene-edited stem cells as a potential treatment for FA!
