Rowan research highlighted in People magazine story of parents' efforts to save baby boys from fatal genetic disease

Rowan research highlighted in People magazine story of parents' efforts to save baby boys from fatal genetic disease

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A gene therapy developed by Dr. Paola Leone, director of the Cell and Gene Therapy Center and a professor at Rowan University School of Osteopathic Medicine, is central to a New York family’s efforts to raise money to save children from a devastatingly fatal childhood disease.

The Landsman family’s GoFundMe campaign, which is profiled in the recent issue of People magazine, focuses on their efforts to find a cure for two of their children who suffer from Canavan disease, a rare, progressive genetic disorder that typically takes a child’s life by age 10.

“Canavan disease is a fatal, inherited disease caused by a genetic mutation,” Dr. Leone explained. “The disease is characterized by progressive and severe brain atrophy that manifests with delayed development, developmental regression, macrocephaly, spasticity, seizures, visual impairment and short life expectancy. There, currently, is no treatment or cure for Canavan disease.”

Dr. Leone leads a team of researchers at Rowan that are developing an innovative therapy that involves surgical delivery of virus-based genetic material into the brains of children with Canavan disease. Pending final federal approval, the therapy will be tested in a clinical trial at the University of Illinois at Chicago at an as yet undetermined date.

The People magazine story, which is available here, details the parents’ efforts to raise funds through a GoFundMe page to support Dr. Leone’s innovative therapy that has shown potential to halt or even reverse the most damaging symptoms of Canavan disease.