Nicolas S. Wein, PhD :: Nationwide Children's Hospital, Columbus, Ohio

Nicolas Wein, PhD

Nicolas  Wein, PhD

Center for Gene Therapy
Principal Investigator

Contact Information

The Research Institute at Nationwide Children's Hospital
700 Children's Drive
Columbus, OH 43205 [ map ]
PH: (614) 355-3771
Email Me

Biography

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 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.

Languages Spoken:

  • English
  • French (Native Speaker)
  • English (Spoken and Written)

Clinical Interests

  • Neuromuscular disorders

Research Interests

Research Center:

Areas of Interest:

  • Our lab 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. Our lab has developed an expertise in translational research, an emerging field which can be summarized as understanding the cause of a disease and learning from it in order to develop innovative therapeutic strategies. We are fully committed to continuing to integrate basic science and gene therapy skills in order to advance understanding and treatment of the muscular dystrophies. Our 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, our lab uses the newest technologies, such as virus-mediated exon-skipping and the CRISPR/Cas9 genome editing tool.

Education and Training

Undergraduate School

  • La Timone Medical School
    Date Completed: 05/30/2006

Graduate School

  • La Timone Medical School
    Date Completed: 12/09/2010

Post Doctoral

  • Center for Gene Therapy at The Research Institute
    Date Completed: 05/31/2016

Department:

  • Center for Gene Therapy

Professional Experience

2009–present

  • Member, World Muscle Society

2006–present

  • Member, American Society of Gene and Cell Therapy

Awards, Honors and Organizations

  • Outstanding Poster Presentation Award, New Directions in Biology and Disease of Skeletal Muscle Conference, Orlando, FL, USA (2016)
  • Outstanding Poster Presentation Award, 19th Annual Meeting of the American Society of Gene and Cell Therapy, Washington, DC, USA (2016)
  • Postdoctoral Scientist of the Year, Nationwide Children's Hospital, Columbus, OH, USA (2016)
  • Young Investigator Prize, New Directions in Biology and Disease of Skeletal Muscle Conference, Chicago, IL, USA (2014)
  • Outstanding Poster Prize, Muscular Dystrophy Association Scientific Meeting, Washington, DC, USA (2013)
  • Poster Award, 17th International Congress of the World Muscle Society, Perth, Australia (2013)
  • Duchenne Research Award for Best Presentation on Therapy of Duchenne Muscular Dystrophy, 18th International Congress of the World Muscle Society, Asilomar, CA, USA (2013)
  • Poster Award, Federative Research Institute 125, Marseille, France (2010).
  • Young Investigator Prices, 15th International Congress of the World Muscle Society, Kumamoto, Japan (2012)
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000