Nicolas Wein

Dr. Nicolas Wein received his Ph.D. in molecular biology in the laboratory of Nicolas Levy, M.D., Ph.D., at the Marseille Medical School in France, where he worked on the development of new diagnosis tools for neuromuscular diseases, the discovery of the Minidysferline and its use for gene transfer, and investigation of the use of exon-skipping for dysferlinopathies. In 2011, he joined the team of Kevin Flanigan, M.D., principal investigator in The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, as a postdoctoral scientist. Dr. Wein worked on the identification of a novel IRES in the dystrophin gene and on an out-of-frame exon-skipping approach that can benefit patients with 5’ mutations in the DMD gene.