Sulforaphane targets multiple pathological processes in Friedreich ataxia patient induced pluripotent stem cells-derived sensory neurons

<p dir="ltr">The thesis investigated the therapeutic potential of a broccoli-derived compound, sulforaphane, for the treatment of an inherited and incurable disease called Friedreich ataxia. The death of cells in the spinal cord, known as sensory neurons, are a key cell type affected in early disease. This study established the molecular profile of genetically diverse sensory neurons derived from patient induced stem cells, and compared the efficacy of sulforaphane with that of the only clinically approved drug, Skyclarys. The study revealed that sulforaphane can improve the viability of sensory neurons while targeting multiple disease processes including the genetic cause, an advantage which Skyclarys lacks. This study has advanced the Friedreich ataxia drug discovery pipeline.</p>