Contact Information Available for logged-in reporters only Newswise STRATFORD 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 (RowanSOM), has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke (NINDS) to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a childs life by age 10. Canavan disease is a fatal, inherited disease caused by a mutation in the aspartaocylase gene, Dr. Leone explained. The disease is characterized by progressive and severe brain atrophy that manifests in delayed development, developmental regression, microcephaly, spasticity, seizures, visual impairment and short life expectancy. There, currently, is no treatment or cure for Canavan disease. Under Dr. Leones direction, a team of RowanSOM researchers and students will examine the potential of stem cells for the treatment of Canavan disease in an animal model. This new study will build on the research teams preliminary data that demonstrated the successful engraftment of stem cells in animal models. Our project will generate pre-clinical data to support the development of a stem-cell based therapy for Canavan … Continue reading
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