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Paola Leone, Ph.D., is a leading researcher of Canavan disease.

Dr. Leone was born and raised in Cagliari, Italy. She received her undergraduate and graduate training in Italy, followed by post-doctoral studies in Montreal and Yale University in New Haven, CT. Her work on Canavan disease started at Yale, where she collaborated with other early pioneers in gene therapy. She left Yale in 1998 to co-direct the (now defunct) Cell & Gene Therapy Center at Thomas Jefferson University in Philadelphia, PA. She now directs The Cell & Gene Therapy Center at the University of Medicine and Dentistry of New Jersey. Current work involves the first gene therapy clinical trial for a neurological degenerative disorder.

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