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Skeletal Muscle Mitochondrial and Autophagic Dysregulation Are Modifiable in Spinal Muscular Atrophy Mikhail, Andrew I.; Ng, Sean Y.; Xhuti, Donald; Lesinski, Magda A.; Chhor, Jennifer; Deguise, Marc-Olivier; De Repentigny, Yves; Nederveen, Joshua P.; Kothary, Rashmi; Tarnopolsky, Mark A.; Ljubicic, Vladimir Share Save
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Identification of the Wnt signal peptide that directs secretion on extracellular vesicles Gurriaran-Rodriguez, Uxia; Datzkiw, David; Radusky, Leandro G.; Esper, Marie; Javandoost, Ehsan; Xiao, Fan; Ming, Hong; Fisher, Solomon; Marina, Alberto; De Repentigny, Yves; Kothary, Rashmi; Azkargorta, Mikel; Elortza, Felix; Rojas, Adriana L.; Serrano, Luis; Hierro, Aitor; Rudnicki, Michael A. Share Save
Liver SMN restoration rescues the Smn spinal muscular atrophy 2B/- mouse model of Sutton, Emma R.; Beauvais, Ariane; Yaworski, Rebecca; De Repentigny, Yves; Reilly, Aoife; de Almeida, Alves; Deguise, Marc-Olivier; Poulin, Kathy L.; Parks, Robin J.; Schneider, Bernard L.; Kothary, Rashmi Share Save
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The systemic complexity of a monogenic disease: the molecular network of spinal muscular atrophy Tapken, Ines; Schweitzer, Theresa; Paganin, Martina; Schuening, Tobias; Detering, Nora T.; Sharma, Gaurav; Niesert, Moritz; Saffari, Afshin; Kuhn, Daniela; Glynn, Amy; Cieri, Federica; Santonicola, Pamela; Cannet, Claire; Gerstner, Florian; Faller, Kiterie M. E.; Huang, Yu-Ting; Kothary, Rashmi; Gillingwater, Thomas H.; Di Schiavi, Elia; Simon, Christian M.; Hensel, Niko; Ziegler, Andreas; Viero, Gabriella; Pich, Andreas; Claus, Peter Share Save
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Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy Alves, Christiano R. R.; Ha, Leillani L.; Yaworski, Rebecca; Sutton, Emma R.; Lazzarotto, Cicera R.; Christie, Kathleen A.; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M.; de la Cruz, Demitri; Maguire, Casey A.; Swoboda, Kathryn J.; Tsai, Shengdar Q.; Kothary, Rashmi; Kleinstiver, Benjamin P. Share Save
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Survival motor neuron protein deficiency alters microglia reactivity Khayrullina, Guzal; Alipio-Gloria, Zaida A.; Deguise, Marc-Olivier; Gagnon, Sabrina; Chehade, Lucia; Stinson, Matthew; Belous, Natalya; Bergman, Elizabeth M.; Lischka, Fritz W.; Rotty, Jeremy; Dalgard, Clifton L.; Kothary, Rashmi; Johnson, Kristen A.; Burnett, Barrington G. Share Save
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SMN Depleted Mice Offer a Robust and Rapid Onset Model of Nonalcoholic Fatty Liver Disease Deguise, Marc-Olivier; Pileggi, Chantal; De Repentigny, Yves; Beauvais, Ariane; Tierney, Alexandra; Chehade, Lucia; Michaud, Jean; Llavero-Hurtado, Maica; Lamont, Douglas; Atrih, Abdelmadjid; Wishart, Thomas M.; Gillingwater, Thomas H.; Schneider, Bernard L.; Harper, Mary-Ellen; Parson, Simon H.; Kothary, Rashmi Share Save
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XIAP Protects Retinal Ganglion Cells in the Mutant ND4 Mouse Model of Leber Hereditary Optic Neuropathy Wassmer, Sarah J.; De Repentigny, Yves; Sheppard, Derek; Lagali, Pamela S.; Fang, Lijun; Coupland, Stuart G.; Kothary, Rashmi; Guy, John; Hauswirth, William W.; Tsilfidis, Catherine Share Save
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