Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment

por Oliveira, Julia Teixeira, Yanick, Christopher, Wein, Nicolas, Gomez Limia, Cintia Elisabeth
Publicado 2023

Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

por Rashnonejad, Afrooz, Amini-Chermahini, Gholamhossein, Taylor, Noah K., Wein, Nicolas, Harper, Scott Q.
Publicado 2020

Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

por Gomez Limia, Cintia, Baird, Megan, Schwartz, Maura, Saxena, Smita, Meyer, Kathrin, Wein, Nicolas
Publicado 2022

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

por Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.
Publicado 2021

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

por Gushchina, Liubov V., Bradley, Adrienne J., Vetter, Tatyana A., Lay, Jacob W., Rohan, Natalie L., Frair, Emma C., Wein, Nicolas, Flanigan, Kevin M.
Publicado 2023

Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients’ Cells

por Barthélémy, Florian, Blouin, Cédric, Wein, Nicolas, Mouly, Vincent, Courrier, Sébastien, Dionnet, Eugénie, Kergourlay, Virginie, Mathieu, Yves, Garcia, Luis, Butler-Browne, Gillian, Lamaze, Christophe, Lévy, Nicolas, Krahn, Martin, Bartoli, Marc
Publicado 2015

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

por Gushchina, Liubov V., Frair, Emma C., Rohan, Natalie, Bradley, Adrienne J., Simmons, Tabatha R., Chavan, Hemantkumar D., Chou, Hsin-Jung, Eggers, Michelle, Waldrop, Megan A., Wein, Nicolas, Flanigan, Kevin M.
Publicado 2021

Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines

por Almeida, Camila F., Robriquet, Florence, Vetter, Tatyana A., Huang, Nianyuan, Neinast, Reid, Hernandez-Rosario, Lumariz, Rajakumar, Dhanarajan, Arnold, W. David, McBride, Kim L., Flanigan, Kevin M., Weiss, Robert B., Wein, Nicolas
Publicado 2023

Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells

por Massouridès, Emmanuelle, Polentes, Jérôme, Mangeot, Philippe-Emmanuel, Mournetas, Virginie, Nectoux, Juliette, Deburgrave, Nathalie, Nusbaum, Patrick, Leturcq, France, Popplewell, Linda, Dickson, George, Wein, Nicolas, Flanigan, Kevin M., Peschanski, Marc, Chelly, Jamel, Pinset, Christian
Publicado 2015

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

por Wein, Nicolas, Vetter, Tatyana A., Vulin, Adeline, Simmons, Tabatha R., Frair, Emma C., Bradley, Adrienne J., Gushchina, Liubov V., Almeida, Camila F., Huang, Nianyuan, Lesman, Daniel, Rajakumar, Dhanarajan, Weiss, Robert B., Flanigan, Kevin M.
Publicado 2022

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

por Gushchina, Liubov V., Vetter, Tatyana A., Frair, Emma C., Bradley, Adrienne J., Grounds, Kelly M., Lay, Jacob W., Huang, Nianyuan, Suhaiba, Aisha, Schnell, Frederick J., Hanson, Gunnar, Simmons, Tabatha R., Wein, Nicolas, Flanigan, Kevin M.
Publicado 2022

X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation

por Barthélémy, Inès, Calmels, Nadège, Weiss, Robert B., Tiret, Laurent, Vulin, Adeline, Wein, Nicolas, Peccate, Cécile, Drougard, Carole, Beroud, Christophe, Deburgrave, Nathalie, Thibaud, Jean-Laurent, Escriou, Catherine, Punzón, Isabel, Garcia, Luis, Kaplan, Jean-Claude, Flanigan, Kevin M., Leturcq, France, Blot, Stéphane
Publicado 2020

A novel DMD IRES results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations

por Wein, Nicolas, Vulin, Adeline, Sofia Falzarano, Maria, Al-Khalili Szigyarto, Christina, Maiti, Baijayanta, Findlay, Andrew, Heller, Kristin N, Uhlén, Mathias, Bakthavachalu, Baskar, Messina, Sonia, Vita, Giuseppe, Passarelli, Chiara, Gualandi, Francesca, Wilton, Steve D, Rodino-Klapac, Louise, Yang, Lin, Dunn, Diane M., Schoenberg, Daniel, Weiss, Robert B., Howard, Michael T., Ferlini, Alessandra, Flanigan, Kevin M.
Publicado 2014