Stem Cells Enable Exploration of Rare Neurological Disease


NYSCF – Robertson Stem Cell Investigator Alumnus Dr. Paul Tesar of Case Western Reserve University School of Medicine collaborated with NYSCF scientists Dr. Valentina Fossati and Dr. Panos Douvaras to explore the varied genetics behind Pelizaeus-Merzbacher Disease, a rare neurological disorder. Using stem cells, the scientists created models of the disease in a dish that displayed different genetic scenarios, identifying individual and shared defects that could inform treatment efforts.

This research, published in The American Journal of Human Genetics, could have a significant impact on clinical approaches to treating this deadly childhood disorder.

Read the paper in The American Journal of Human Genetics

Read the press release on EurekAlert

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