Nicolas Wein

Background

About Nicolas Wein

I received his PhD in Molecular Biology in the laboratory of Nicolas Levy, MD, PhD, 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, I joined the team of Kevin Flanigan, MD, principal investigator in The Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital, as a postdoctoral scientist. I 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. My lab now focuses on neuromuscular disorders such as Duchenne Muscular dystrophy, which is a devastating disease leading to death of patients at 20-30 years of age. The lab long-term projects focus on RNA and DNA editing and gene transfer as therapies for neuromuscular disorders, but they will also include more fundamental research on muscle function. In particular, we are using the newest technologies, such as virus-mediated exon-skipping and the CRISPR/Cas9 genome editing tool.