Contact Information

Call us at: (614)355-3771

Email Nicolas S Wein, PhD

Center for Gene TherapyAbigail Wexner Research Institute700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Nicolas S Wein

Research

Lab(s)

Center for Gene Therapy

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.

View My Publications

Publications

                  Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.

                


                  Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.

                


                  Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 

                


                  Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.

                


                  Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  

                


                  Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.

                


                  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 Nov; 32: 1346-1359.

                


                  Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.

                


                  Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.

                


                  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;  

                


                  Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.

                


                  Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.

                


                  Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  

                


                  Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

Biography

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Primary Department

Center for Gene Therapy

Awards, Honors & 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, 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, 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, 2013 Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012 Poster Award, Federative Research Institute 125, Marseille, France, 2010 Member, World Muscle Society, 2009 - Present Member, American Society of Gene and Cell Therapy, 2006 - Present

Education

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Clinical Interests

Neuromuscular disorders

Professional Experience

2009 - Present World Muscle Society, Member2006 - Present American Society of Gene and Cell Therapy, Member

Contact Information

Center for Gene Therapy

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Call us at: (614)355-3771

Email Nicolas S Wein, PhD

Center for Gene TherapyAbigail Wexner Research Institute700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Nicolas S Wein

Research

Lab(s)

Center for Gene Therapy

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.

View My Publications

Publications

                  Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.

                


                  Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.

                


                  Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 

                


                  Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.

                


                  Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  

                


                  Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.

                


                  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 Nov; 32: 1346-1359.

                


                  Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.

                


                  Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.

                


                  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;  

                


                  Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.

                


                  Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.

                


                  Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  

                


                  Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

Biography

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Primary Department

Center for Gene Therapy

Awards, Honors & 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, 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, 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, 2013 Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012 Poster Award, Federative Research Institute 125, Marseille, France, 2010 Member, World Muscle Society, 2009 - Present Member, American Society of Gene and Cell Therapy, 2006 - Present

Education

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Clinical Interests

Neuromuscular disorders

Professional Experience

2009 - Present World Muscle Society, Member2006 - Present American Society of Gene and Cell Therapy, Member

Contact Information

Center for Gene Therapy

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Call us at: (614)355-3771

Email Nicolas S Wein, PhD

Center for Gene TherapyAbigail Wexner Research Institute700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Nicolas S Wein

Contact Information

  • Call us at:
  • (614)355-3771
  • Email Nicolas S Wein, PhD
  • Center for Gene TherapyAbigail Wexner Research Institute700 Children’s DriveColumbus, OH 43205 (map)

Learn more about Nicolas S Wein

Research

Lab(s)

Center for Gene Therapy

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.

View My Publications

Publications

                  Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.

                


                  Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.

                


                  Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 

                


                  Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.

                


                  Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  

                


                  Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.

                


                  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 Nov; 32: 1346-1359.

                


                  Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.

                


                  Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.

                


                  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;  

                


                  Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.

                


                  Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.

                


                  Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  

                


                  Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

Research

Lab(s)

Center for Gene Therapy

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.

View My Publications

Publications

                  Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.

                


                  Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.

                


                  Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 

                


                  Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.

                


                  Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  

                


                  Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.

                


                  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 Nov; 32: 1346-1359.

                


                  Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.

                


                  Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.

                


                  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;  

                


                  Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.

                


                  Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.

                


                  Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  

                


                  Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

Research

Lab(s)

Center for Gene Therapy

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.

View My Publications

Publications

                  Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.

                


                  Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.

                


                  Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 

                


                  Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.

                


                  Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  

                


                  Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.

                


                  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 Nov; 32: 1346-1359.

                


                  Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.

                


                  Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.

                


                  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;  

                


                  Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.

                


                  Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.

                


                  Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  

                


                  Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

Lab(s)

Center for Gene Therapy

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.

View My Publications

Publications

                  Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.

                


                  Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.

                


                  Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 

                


                  Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.

                


                  Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  

                


                  Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.

                


                  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 Nov; 32: 1346-1359.

                


                  Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.

                


                  Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.

                


                  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;  

                


                  Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.

                


                  Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.

                


                  Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  

                


                  Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

Lab(s)

Center for Gene Therapy

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.

Lab(s)

Center for Gene Therapy

  • Center for Gene Therapy

  • View My Publications

                    Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.



                Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.



                Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12: 



                Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.



                Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;  



                Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.



                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 Nov; 32: 1346-1359.



                Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.



                Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.



                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;  



                Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.



                Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.



                Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;  



                Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

View More Publications

  • Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy. Mol Ther Nucleic Acids. 2022 Dec 13; 30: 479-492.
  • Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy. Cell Rep. 2022 Dec 6; 41: 111751.
  • Gomez Limia C, Baird M, Schwartz M, Saxena S, Meyer K, Wein N. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. J Pers Med. 2022 Nov 30; 12:
  • Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse. Mol Ther Methods Clin Dev. 2022 Sep 8; 26: 279-293.
  • Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Intron Mutations and Early Transcription Termination in Duchenne and Becker muscular dystrophy. Hum Mutat. 2022 Feb 14;
  • Lesman D, Rodriguez Y, Rajakumar D, Wein N. U7 snRNA, a Small RNA with a Big Impact in Gene Therapy. Hum Gene Ther. 2021 Nov; 32: 1317-1329.
  • 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 Nov; 32: 1346-1359.
  • Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping. Hum Gene Ther. 2021 Sep; 32: 882-894.
  • Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping. Mol Ther Methods Clin Dev. 2021 Jun 11; 21: 325-340.
  • 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;
  • Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes. Mol Ther Nucleic Acids. 2021 Mar 5; 23: 476-486.
  • Barthélémy I, Calmels N, Weiss RB, Tiret L, Vulin A, Wein N, Peccate C, Drougard C, Beroud C, Deburgrave N, Thibaud JL, Escriou C, Punzón I, Garcia L, Kaplan JC, Flanigan KM, Leturcq F, Blot S. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation. Skelet Muscle. 2020 Aug 7; 10: 23.
  • Barthélémy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscul Disord. 2018 Jul 26;
  • Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach. J Neuromuscul Dis. 2017; 4: 199-207.

Biography

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Biography

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Biography

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Nicolas Wein, PhD, is a principal investigator in the Center for Gene Therapy and an assistant professor of Pediatrics at The Ohio State University. He received his doctorate degree 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 minidysferlin gene 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, at the Center for Gene Therapy as a postdoctoral scientist, where he worked the identification and therapeutic implications of a novel IRES in the dystrophin gene. He established his own laboratory as an independent investigator at in the center in 2016.

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Primary Department

Center for Gene Therapy

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Primary Department

Center for Gene Therapy

Academic and Clinical Areas

Center for Gene Therapy

Principal Investigator

Primary Department

Center for Gene Therapy

Center for Gene Therapy

Principal Investigator

Primary Department

Center for Gene Therapy

  • Center for Gene Therapy
  • Principal Investigator
  • Primary Department
  • Center for Gene Therapy

Awards, Honors & 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, 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, 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, 2013 Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012 Poster Award, Federative Research Institute 125, Marseille, France, 2010 Member, World Muscle Society, 2009 - Present Member, American Society of Gene and Cell Therapy, 2006 - Present

Awards, Honors & 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, 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, 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, 2013 Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012 Poster Award, Federative Research Institute 125, Marseille, France, 2010 Member, World Muscle Society, 2009 - Present Member, American Society of Gene and Cell Therapy, 2006 - Present

Awards, Honors & 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, 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, 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, 2013 Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012 Poster Award, Federative Research Institute 125, Marseille, France, 2010 Member, World Muscle Society, 2009 - Present Member, American Society of Gene and Cell Therapy, 2006 - Present

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, 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, 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, 2013 Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012 Poster Award, Federative Research Institute 125, Marseille, France, 2010 Member, World Muscle Society, 2009 - Present Member, American Society of Gene and Cell Therapy, 2006 - Present

  • 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, 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, 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, 2013
  • Young Investigator Prize, 15th International Congress of the World Muscle Society, Kumamoto, Japan, 2012
  • Poster Award, Federative Research Institute 125, Marseille, France, 2010
  • Member, World Muscle Society, 2009 - Present
  • Member, American Society of Gene and Cell Therapy, 2006 - Present

Education

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Education

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Education

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Post Doctoral

Center for Gene Therapy at The Research Institute

Date Completed: 05/31/2016

Graduate School

La Timone Medical School

Date Completed: 12/09/2010

Undergraduate School

La Timone Medical School

Date Completed: 05/30/2006

Clinical Interests

Neuromuscular disorders

Clinical Interests

Neuromuscular disorders

Clinical Interests

Neuromuscular disorders

Neuromuscular disorders

  • Neuromuscular disorders

Professional Experience

2009 - Present World Muscle Society, Member2006 - Present American Society of Gene and Cell Therapy, Member

Professional Experience

2009 - Present World Muscle Society, Member2006 - Present American Society of Gene and Cell Therapy, Member

Professional Experience

2009 - Present World Muscle Society, Member2006 - Present American Society of Gene and Cell Therapy, Member

2009 - Present World Muscle Society, Member2006 - Present American Society of Gene and Cell Therapy, Member

2009 - Present World Muscle Society, Member

Contact Information

Center for Gene Therapy

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Center for Gene Therapy

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Contact Information

Center for Gene Therapy

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Center for Gene Therapy

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)

Call us at: (614)355-3771

Email Nicolas Wein

                    Abigail Wexner Research Institute700 Children's DriveColumbus, OH 43205 (map)
  • Call us at:
  • (614)355-3771
  • Email Nicolas Wein
  • Abigail Wexner Research Institute700 Children’s DriveColumbus, OH 43205 (map)