Rowan Researcher Targets Stem Cell-Based Therapy for Rare Childhood Disease

Paola Leone, PhD, the director of the Cell and Gene Therapy Center and a professor of Cell Biology at the Rowan University School of Osteopathic Medicine , has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a child's life by age 10. "Canavan disease is a fatal, inherited disease caused by a mutation in the aspartaocylase gene," Dr. Leone explained.

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