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

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Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes von Rashnonejad, Afrooz, Amini-Chermahini, Gholamhossein, Taylor, Noah K., Wein, Nicolas, Harper, Scott Q.

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Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy von Waldrop, Megan A., Moore, Steven A., Mathews, Katherine D., Darbro, Benjamin W., Medne, Livja, Finkel, Richard, Connolly, Anne M., Crawford, Thomas O., Drachman, Daniel, Wein, Nicolas, Habib, Ali A., Krzesniak‐Swinarska, Monika A., Zaidman, Craig M., Collins, James J., Jokela, Manu, Udd, Bjarne, Day, John W., Ortiz‐Guerrero, Gloria, Statland, Jeff, Butterfield, Russell J., Dunn, Diane M., Weiss, Robert B., Flanigan, Kevin M.

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

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Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45 von Findlay, Andrew R., Wein, Nicolas, Kaminoh, Yuuki, Taylor, Laura E., Dunn, Diane M., Mendell, Jerry R., King, Wendy M., Pestronk, Alan, Florence, Julaine M., Mathews, Katherine D., Finkel, Richard S., Swoboda, Kathryn J., Howard, Michael T., Day, John W., McDonald, Craig, Nicolas, Aurélie, Le Rumeur, Elisabeth, Weiss, Robert B., Flanigan, Kevin M.

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Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping von Wein, Nicolas, Avril, Aurélie, Bartoli, Marc, Beley, Cyriaque, Chaouch, Soraya, Laforêt, Pascal, Behin, Anthony, Butler-Browne, Gillian, Mouly, Vincent, Krahn, Martin, Garcia, Luis, Lévy, Nicolas

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

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

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

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

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X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation von Barthelemy, Ines, Calmels, Nadege, Weiss, Robert B., Tiret, Laurent, Vulin, Adeline, Wein, Nicolas, Peccate, Cecile, Drougard, Carole, Beroud, Christophe, Deburgrave, Nathalie, Thibaud, Jean-Laurent, Escriou, Catherine, Punzon, Isabel, Garcia, Luis, Kaplan, Jean-Claude, Flanigan, Kevin M., Leturcq, France, Blot, Stephane

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The ZZ Domain of Dystrophin in DMD: Making Sense of Missense Mutations von Vulin, Adeline, Wein, Nicolas, Strandjord, Dana M., Johnson, Eric K., Findlay, Andrew R., Maiti, Baijayanta, Howard, Michael T., Kaminoh, Yuuki J., Taylor, Laura E., Simmons, Tabatha R., Ray, Will C., Montanaro, Federica, Ervasti, Jim M., Flanigan, Kevin M.

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Personalized gene and cell therapy for Duchenne Muscular Dystrophy von Barthélémy, Florian, Wein, Nicolas

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U7 snRNA, a Small RNA with a Big Impact in Gene Therapy von Lesman, Daniel, Rodriguez, Yacidzohara, Rajakumar, Dhanarajan, Wein, Nicolas

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Performance Requirements of Membrane Reactors for the Application in Renewable Methanol Synthesis: A Techno‐Economic Assessment von Dieterich, Vincent, Wein, Nicolas, Spliethoff, Hartmut, Fendt, Sebastian

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Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications von Wein, Nicolas, Dunn, Diane M., Waldrop, Megan A., Gushchina, Liubov, Frair, Emma C., Weiss, Robert B., Flanigan, Kevin M.

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

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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates von Powers, Samantha, Likhite, Shibi, Gadalla, Kamal K., Miranda, Carlos J., Huffenberger, Amy J., Dennys, Cassandra, Foust, Kevin D., Morales, Pablo, Pierson, Christopher R., Rinaldi, Federica, Perry, Stephanie, Bolon, Brad, Wein, Nicolas, Cobb, Stuart, Kaspar, Brian K., Meyer, Kathrin C.

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Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice von Wein, Nicolas, Vulin, Adeline, Falzarano, Maria S, Szigyarto, Christina Al-Khalili, Maiti, Baijayanta, Findlay, Andrew, Heller, Kristin N, Uhlén, Mathias, Bakthavachalu, Baskar, Messina, Sonia, Vita, Giuseppe, Passarelli, Chiara, Passarelli, Simona, Bovolenta, Matteo, Neri, Marcella, Gualandi, Francesca, Wilton, Steve D, Rodino-Klapac, Louise R, Yang, Lin, Dunn, Diane M, Schoenberg, Daniel R, Weiss, Robert B, Howard, Michael T, Ferlini, Alessandra, Flanigan, Kevin M

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

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