Nicolas WEIN

• mRNA Processing
• Gene Therapy
• Lentivirus
• Adeno and Adeno-associated viruses
• Adeno-associated virus mediated gene therapy to treat neurological and neuromuscular disorders

Education:

• 2025-Present Associate Professor, CPJ, TaRGeT, UMR1089, Nantes University, Nantes, France.
• 2023-2025 Associate Professor, Departments of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
• 2016-2023 Assistant Professor, Departments of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
• 2016-2025 Principal Investigator, Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, Ohio, USA.
• 2016-2025 Posdoctoral Scientist, Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, Ohio, USA.
• 2006 - 2010 PhD Student in the Medical Genetics and Functional Genomics. Marseille, France.

Selected publications (among 39 publications, H-index 19)

• Gushchina LV, Bradley AJ, Vetter TA, Lay JW, Rohan NL, Frair EC, Wein N, Flanigan KM. Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA- mediated therapy in the Dup2 mouse model. Mol Ther Methods Clin Dev. 2023 Oct 26;31:101144.
• Camila F. Almeida, Florence Robriquet, Daniel Lesman, Nianyuan Huang, Hsin-Jung Chou, Dhanarajan Rajakumar, Reid Neinast, W. David Arnold, Kim L. McBride, Nicolas Wein. Direct conversion of skin fibroblasts into myotubes to model several CTG repeat expansion causing myotonic dystrophy type 1. Front Cell Dev Biol. 2023 Jun 15;11:1181040.
• Nicolas Wein, Tatyana A. Vetter, Adeline Vulin, Tabatha R. Simmons, Emma C. Frair, Adrienne Bradley, Liubov V. Gushchina, Camila F. Almeida, Nianyuan Huang, Daniel Lesman, Dhanarajan Rajakumar, Robert B. Weiss, Kevin M. Flanigan. Systemic Delivery of an AAV9 Exon Skipping Vector Significantly Improves or Prevents Features of Duchenne Muscular Dystrophy in the Dup2 Mouse Model. Molecular Therapy - Methods & Clinical Development (Volume 26) - August 3, 2022.
• Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Aug 26.
• Wein N, Dunn DM, Waldrop MA, Gushchina LV, Frair EC, Weiss RB, Flanigan KM. Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications. Hum Gene Ther. 2021 Jul 20.
• Almeida CF, Frair EC, Huang N, Neinast R, McBride KL, Weiss RB, Flanigan KM, Wein N. Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders. J Vis Exp. 2021 Apr 3;(170).
• Findlay A.; Wein N., Kaminoh K; Taylor L.; Dunn D., Mendell J.; King W.; Pestronk A.; Florence J.; Mathews K.; Finkel R.; Swoboda K.; Howard M.; Day J.; McDonald C.; Weiss R.,Flanigan K. for the United Dystrophinopathy Project. Clinical phenotypes as predictors of the outcome of DMD exon 45 skipping. Ann Neurol. 2015 Apr;77(4):668-74.
• Wein N, Vulin A, Falzarano MS, Szigyarto A, Maiti B, Findlay A, Uhlen M, Bakthavachalu B, Messina S, Kaminoh Y, Vattemi G, Perrone D, Taylor L, Gualandi F, Weiss RB, Schoenberg D, Howard M, Ferlini A, Flanigan KM. Using out of frame exon skipping to induce IRES-driven expression of an N-truncated dystrophin isoform: A novel therapeutic approach to mutations in the 5’ region of DMD. Nat Med. 2014 Sep;20(9):992-1000.
• Wein N*, Krahn M*, Bartoli M*, Lostal W, Courrier S, Bourg-Alibert N, Nguyen K, Vial C, Streichenberger N, Labelle V, DePetris D, Pécheux C, Leturcq F, Cau P, Richard I and Lévy N. A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy. Sci Transl Med. 2010 Sep 22;2(50):50ra69.
• Wein N, Avril A, Bartoli M, Beley C, Chaouch S, Laforêt P, Behin A, Butler-Browne G, Mouly V, Krahn M, Garcia L, Lévy N. Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping. Hum Mutat. 2010 Feb;31(2):136-42.

Scholastic Honors (among 12):

• Excellence in Undergraduate Research Mentoring award at The Ohio State University in Columbus, OH, USA (2021); the Outstanding RINCH Team Award (2017)
• Postdoctoral Scientist of the Year at Nationwide Children’s Hospital (2014) in, Columbus, OH, USA
• Young Investigator Prize at New Direction in Biology and Disease of Skeletal Muscle in Chicago, IL, USA (2014)
• Duchenne Research Award at the 18th World Muscle Society in Asilomar, CA, USA (2013)

Mentoring /Teaching

I have also been fortunate to supervise several undergraduate and graduate students (>15) during both my graduate school, my postdoctoral time and Assistant and Associate Professor. my students since I opened my lab won 23 prestigious awards (23) including the prestigious Rhodes Scholarship

• Teaching Experiences

I taught 25 classes for Undergraduates, PhD students, MD/PhD students at The Ohio State University (OSU) and Nationwide Children’s Hospital in Columbus, OH, USA for which I received Outstanding evaluations (91%). I also gave 8 educational sessions taught at national and international events and organized 4 courses/educational events:

• Participation of the creation of PEDS5088 and PED5077
• Member of the Education Committee member of the American Society of Gene and Cell Therapy (ASGCT) since 2016.