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Nicolas S. Wein, PhD
Center for Gene Therapy
Abigail Wexner Research Institute
700 Children's Drive
Columbus, Ohio, United States
Dr. Nicolas S. Wein is a Principal Investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute. Dr. Wein 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, he 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. Dr. Wein worked on the identification of 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.
AAV-Based Gene Therapies for the Muscular DystrophiesMuscular dystrophy is a group of progres...
NCATS, FDA Co-Host Workshop on Gene Therapy for Rare Diseases“We are now at the point of translatin...
First targeted treatment success for Duchenne muscular dystrophyhttps://www.youtube.com/watch?v=tkxTn46_...
Why my patient advocacy organization is investing in CRISPRIn last year’s action film “Rampage,...
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyDuchenne muscular dystrophy (DMD) is a p...
Gene Therapy as a Potential Therapeutic Option for Duchenne Muscular Dystrophy: A Qualitative Preference Study of Pa...Objectives: Duchenne muscular dystrophy...
Gene Therapy OverviewIn this brief document, we want to expla...