Recharging the Batteries

The Mission

Combating rare mitochondrial diseases using innovative stem cell therapy.

The challenge

Mitochondrial diseases with an incidence of 1 in 10.000 are devastating metabolic diseases which can cause a variety of clinical manifestations. They entail a pathological loss of energy, which generally affects organs with high energy demands, such as muscles and nerves. As no cure for these diseases is available, we want to explore a novel therapeutic strategy to develop stem cells that can transfer healthy mitochondria to affected tissues to provide sufficient energy and ameliorate their clinical symptoms. As proof, we will test this for the optic nerve, which is damaged in patients with Leber Hereditary Optic Neuropathy (LHON).

The solution

Our solution is the transfer of healthy mitochondria to damaged tissue, using stem cells as suppliers. The mitochondria will be transferred through nanotubes, made by the stem cells. This method has been successfully applied to transfer mitochondria in other diseases. We will apply it specifically to Leber's Hereditary Optic Neuropathy (LHON), which is characterised by loss of vision due to a mitochondrial dysfunction in the optic nerve. Our stem cells will sense the damage and provide the optic nerve with new mitochondria. We will test our method in vitro, using retinal ganglion cells and mesenchymal stem cells.