Showing 1 - 7 results of 7 for search 'Nicolas Wein', query time: 0.03s Refine Results
  1. 1
    Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment

    Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment by Julia Teixeira Oliveira, Christopher Yanick, Nicolas Wein, Nicolas Wein, Cintia Elisabeth Gomez Limia

    Published 2023-10-01
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  2. 2
    Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

    Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders by Cintia Gomez Limia, Megan Baird, Maura Schwartz, Smita Saxena, Kathrin Meyer, Nicolas Wein

    Published 2022-11-01
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  3. 3
    Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

    Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping by Tabatha R. Simmons, Tatyana A. Vetter, Nianyuan Huang, Adeline Vulin-Chaffiol, Nicolas Wein, Kevin M. Flanigan

    Published 2021-06-01
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  4. 4
    Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines

    Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines by Camila F. Almeida, Florence Robriquet, Tatyana A. Vetter, Nianyuan Huang, Reid Neinast, Lumariz Hernandez-Rosario, Dhanarajan Rajakumar, W. David Arnold, W. David Arnold, Kim L. McBride, Kim L. McBride, Kevin M. Flanigan, Kevin M. Flanigan, Kevin M. Flanigan, Robert B. Weiss, Nicolas Wein, Nicolas Wein

    Published 2023-06-01
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  5. 5
    Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

    Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model by Liubov V. Gushchina, Adrienne J. Bradley, Tatyana A. Vetter, Jacob W. Lay, Natalie L. Rohan, Emma C. Frair, Nicolas Wein, Kevin M. Flanigan

    Published 2023-12-01
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  6. 6
    Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

    Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse by Nicolas Wein, Tatyana A. Vetter, Adeline Vulin, Tabatha R. Simmons, Emma C. Frair, Adrienne J. Bradley, Liubov V. Gushchina, Camila F. Almeida, Nianyuan Huang, Daniel Lesman, Dhanarajan Rajakumar, Robert B. Weiss, Kevin M. Flanigan

    Published 2022-09-01
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  7. 7
    Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

    Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy by Liubov V. Gushchina, Tatyana A. Vetter, Emma C. Frair, Adrienne J. Bradley, Kelly M. Grounds, Jacob W. Lay, Nianyuan Huang, Aisha Suhaiba, Frederick J. Schnell, Gunnar Hanson, Tabatha R. Simmons, Nicolas Wein, Kevin M. Flanigan

    Published 2022-12-01
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