Mohammad Rafi received his PhD in Animal Biology from the University of Montpellier, France, in 1970. Though he has worked on several lysosomal storage diseases, his main research interest is gene therapy of neurodegenerative disorders using animal models of globoid cell leukodystrophy (Krabbe disease). With successful AAVrh10-mediated treatment of murine and canine models, his research is moving towards the treatment trials of human patients.