Research / Clinical Summary

Theodore Friedmann, MD Professor, Pediatrics Tumor Growth, Invasion & Metastasis Program Contact by Email

My laboratory is studying the development and possible new treatment concepts for severe neurological disorders in children, including a genetic pediatric disease (Lesch Nyhan DIsease-LND) that causes compulsive self-injurious behavior.

The cause of the disease is a deficiency of the neurotransmitter dopamine, a deficiency very similar to that of the adult disease Parkinson's disease. Studies with LND therefore offer an important route toward understanding a number of brain developmental and degenerative diseases.

We are examining the ways in which stem cells decide to become dopamine neurons in the brain and how the defect in LND disrupts that normal process.