Nicolas Wein, PhD

Nicolas Wein, is a PhD who received his doctorate degree in Molecular Biology in the laboratory of Nicolas Levy, MD, PhD, at the Marseille Medical School in France. He worked on new diagnosis tools and therapy for dysferlinopathies. In 2011, he did a postdoctoral position at the Center for Gene Therapy, in columbus, Ohio, USA, where he developed a therapy for a subpopulation of Duchenne Muscular Dystrophy patients (DMD; NCT04240314). In 2016, he opened his own laboratory as an independent investigator and became an associate professor in 2023. His laboratory studies the mechanisms of neuromuscular disorders and the development of treatment for these diseases with a primary focus on DMD. Dr. Wein recently joined Cure Rare Disease organization independently as a consultant to provide scientific guidance to help developing treatment for patient affected by neuromuscular disorders. His experiences in exon skipping and gene therapy will help CRD missions and goals.