During my PhD with Pr Nicolas Levy (University of Marseille, France), I initiated an innovative translational project comprising an RNA-editing adeno-associated viral vector (AAV) gene therapy for limb-girdle muscular dystrophy. My PhD resulted in a Science Translational Medicine and 10 additional publications (F. Barthélémy et al., 2011, 2015; Bartoli et al., 2012; Blandin et al., 2012; Krahn et al., 2009, 2010; Lévy et al., 2010; Wein, Avril, et al., 2010a, 2010b; Wein, Krahn, et al., 2010a, 2010b).
In 2011, I started my postdoctoral position in Dr. Flanigan's lab at NationWide Children's Hospital, in Columbus, Ohio in the USA, developing gene therapy for Duchenne Muscular Dystrophy (DMD). I identified a novel internal-ribosome-entry-site (IRES) within the DMD gene and applied my Ph.D. expertise in RNA AAV gene therapy to develop a therapeutic approach using vectorized exon skipping (VES): one published in Nature Medicine as a 1st author, work that led to a clinical trial in 2020 and 5 additional first or second-author publications.(Findlay et al., 2015; Flanigan et al., 2013; Massouridès et al., 2015; Powers et al., 2023; Vulin et al., 2014, 2015; Wein et al., 2014, 2015, 2017, 2022)
Career summary
In 2010, Dr. Nicolas 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 discovery of the Minidysferlin 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, in The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital (NCH), as a postdoctoral scientist. Thanks to his postdoctoral work, he, Drs. Flanigan and Waldrop initiated a clinical trial for a subpopulation of DMD patients in early 2020. In 2016, he opened his lab and continued working on neuromuscular disorders and neurological disorders. His lab continues to integrate basic science and gene therapy in order to advance understanding and treatment of the neurological and muscular disorders. His team long-term projects focus on RNA and DNA editing and gene transfer as therapies using adeno-associated virus and U7snRNA, a small nuclear RNA (RNA editing tool) for neuromuscular and neurological disorders. His team is also investigating alternative way to target both central nervous system and muscles by using alternative delivery of AAV. In 2025, Dr. Wein got a Chair d’excellence at INSERM and decided to move to France, in Nantes at the TaRGeT institute at UMR1089/University of Nantes.
Overall, Dr. Wein acquired 19 funded grants (nine as PI), published 21 manuscripts since 2016. His H-index is 22 (Google Scholar) or 19 (Scopus). He was fortunate to supervise several technicians, undergraduates, graduate students and postdocs (>15). He developed strong mentorship and teaching skills, which resulted in several prestigious awards that he and his students obtained since he opened his lab: 5 best postdoc presentations (Drs. De Freitas and Rajakumar; 7 awards or fellowships for Daniel Lesman, a graduate student (Outstanding first year student award; Biomedical Science Research Grant; Ted R. Robinson Scholarship; STEP Enrichment grant; Ohio State Goldwater Scholarship, Astronaut Scholarship, and most notably, a Rhodes Scholarship); 4 national and international Fellowships (Theo Charnay (Marseille Medical School), Kaya Ceyhan, Justine Guguin (Ecole Normale Supérieure, Lyon); Charlene Lopez (Universite de Toulouse). He also taught 25 classes for Undergraduates, PhD students, MD/PhD students at The Ohio State University (OSU) and NCH in Columbus, OH, USA for which he received Outstanding evaluations (91%). He also gave 8 educational sessions taught at national and international events and organised 4 courses/educational events. He is also a member of the Education Committee member of the American Society of Gene and Cell Therapy (ASGCT) since 2016. He also participated in 5 national and international committees, 20 organisational activities at national meetings, 4 Thesis committees, 11 invited talks nationally and internationally, 18 talks at national/international conferences since 2016 and am reviewing for 17 different journals and 10 patient organisations.
Dr. Wein has been a member of the the World Muscle Society since 2009, and has received several awards during all these years. He benefited from all of that and wants to give back to the WMS community by helping the board.
What is the personal or professional achievement you’re most proud of?
Since my master degree, i am proud to have always be connected with patients despite not being an M.D. I am also proud to have been able to bring a therapy to clinic and mentored so many students.
Why did you first join the WMS?
I first joined the WMS during my PhD. I really love this community, it composed by MD and PhD and all of them are very collaborative and dedicated to help patients.
What is your goal in joining the Executive Board?
After being for so many year with the WMS, benefiting from awards, I would like to help the WMS community and while I am already involved with for example the WMS website and reviewing abstracts for WMS, I would like to contribute to this amazing community. I have been interacting with so many great researchers from Pr Dubowitz, Pr Burghes, Dr Wilton, Pr Straub, Pr Servais and Pr Bonne and I feel it's time for me to be even more involved with this community.
