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Researchers

Nicolas S. Wein, PhD

Researcher
Principal Investigator
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.

 

Representative Publications:

Personalized gene and cell therapy for Duchenne Muscular Dystrophy

The First Exon Duplication Mouse Model of Duchenne Muscular Dystrophy: A Tool for Therapeutic Development

Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy

Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach

Clinical Phenotypes as Predictors of the Outcome of Skipping Around DMD Exon 45