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Profilin1 is required for prevention of mitotic catastrophe in murine and human glomerular diseases Tian, Xuefei; Pedigo, Christopher E.; Li, Ke; Ma, Xiaotao; Bunda, Patricia; Pell, John; Lek, Angela; Gu, Jianlei; Zhang, Yan; Rangel, Paulina X. Medina; Li, Wei; Schwartze, Eike; Nagata, Soichiro; Lerner, Gabriel; Perincheri, Sudhir; Priyadarshini, Anupama; Zhao, Hongyu; Lek, Monkol; Menon, Madhav C.; Fu, Rongguo; Ishibe, Shuta Share Save
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy Lek, Angela; Wong, Brenda; Keeler, Allison; Blackwood, Meghan; Ma, Kaiyue; Huang, Shushu; Sylvia, Katelyn; Batista, A. Rita; Artinian, Rebecca; Kokoski, Danielle; Parajuli, Shestruma; Putra, Juan; Carreon, C. Katte; Lidov, Hart; Woodman, Keryn; Pajusalu, Sander; Spinazzola, Janelle M.; Gallagher, Thomas; Larovere, Joan; Balderson, Diane; Black, Lauren; Sutton, Keith; Horgan, Richard; Lek, Monkol; Flotte, Terence Share Save
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The Muscular Dystrophy Association's neuroMuscular ObserVational Research Data Hub (MOVR): Design, Methods, and Initial Observations Kilroy, Elisabeth A.; Burris, Rachael; Javelosa, Edritz; Waits, Jessica; Lek, Angela; Rodgers, Rayne; Opgenorth, Hayley; Hesterlee, Sharon Share Save
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Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy Lek, Angela; Zhang, Yuanfan; Woodman, Keryn G.; Huang, Shushu; DeSimone, Alec M.; Cohen, Justin; Ho, Vincent; Conner, James; Mead, Lillian; Kodani, Andrew; Pakula, Anna; Sanjana, Neville; King, Oliver D.; Jones, Peter L.; Wagner, Kathryn R.; Lek, Monkol; Kunkel, Louis M. Share Save
Transgenic zebrafish model of DUX4 misexpression reveals a developmental role in FSHD pathogenesis Pakula, Anna; Lek, Angela; Widrick, Jeffrey; Mitsuhashi, Hiroaki; Gwilt, Katlynn M. Bugda; Gupta, Vandana A.; Rahimov, Fedik; Criscione, June; Zhang, Yuanfan; Gibbs, Devin; Murphy, Quinn; Manglik, Anusha; Mead, Lillian; Kunkel, Louis Share Save
A limb-girdle muscula dystrophy 21 model of muscular dystrophy identifies corrective drug compounds for dystroglycanopathies Serafini, Peter R.; Feyder, Michael J.; Hightower, Rylie M.; Garcia-Perez, Daniela; Vieira, Natassia M.; Lek, Angela; Gibbs, Devin E.; Moukha-Chafiq, Omar; Augelli-Szafran, Corinne E.; Kawahara, Genri; Widrick, Jeffrey J.; Kunkel, Louis M.; Alexander, Matthew S. Share Save
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SMCHD1 mutations associated with a rare muscular dystrophy can also cause isolated arhinia and Bosma arhinia microphthalmia syndrome Shaw, Natalie D.; Brand, Harrison; Kupchinsky, Zachary A.; Bengani, Hemant; Plummer, Lacey; Jones, Takako I.; Erdin, Serkan; Williamson, Kathleen A.; Rainger, Joe; Stortchevoi, Alexei; Samocha, Kaitlin; Curra, Benjamin B.; Dunican, Donncha S.; Collins, Ryan L.; Willer, Jason R.; Lek, Angela; Lek, Monkol; Nassan, Malik; Pereira, Shahrin; Kammin, Tammy; Lucente, Diane; Silva, Alexandra; Seabra, Catarina M.; Chiang, Colby; Ana, Yu; Ansari, Morad; Rainger, Jacqueline K.; Joss, Shelagh; Smith, Jill Clayton; Lippincott, Margaret F.; Singh, Sylvia S.; Patel, Nirav; Jing, Jenny W.; Law, Jennifer R.; Ferraro, Nalton; Verloes, Main; Rauch, Anita; Steindl, Katharina; Zweier, Markus; Scheer, Ianina; Sato, Daisuke; Okamoto, Nobuhiko; Jacobsen, Christina; Tryggestad, Jeanie; Chernausek, Steven; Schimmenti, Lisa A.; Brasseur, Benjamin; Cesaretti, Claudia; Garcia-Ortiz, Jose E.; Pineda Buitrago, Tatiana; Perez Silva, Orlando; Hoffman, Jodi D.; Muehlbauer, Wolfgang; Ruprecht, Klaus W.; Loeys, Bart L.; Shino, Masato; Kaind, Angela M.; Cho, Chie-Hee; Morton, Cynthia C.; Meehan, Richard R.; van Heyningen, Veronica; Liao, Eric C.; Balasubramanian, Ravikumar; Hall, Janet E.; Seminara, Stephanie B.; Macarthur, Daniel; Moore, Steven A.; Yoshiura, Koh-ichiro; Gusella, James F.; Marsh, Joseph A.; Graham, John M., Jr.; Lin, Angela E.; Katsanis, Nicholas; Jones, Peter L.; Crowley, William F., Jr.; Davis, Erica E.; FitzPatrick, David R.; Talkowski, Michael E. Share Save
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MicroRNA-486 dependent modulation of DOCK3/PTEN/AKT signaling pathways improves muscular dystrophy-associated symptoms Alexander, Matthew S.; Carlos Casar, Juan; Motohashi, Norio; Vieira, Natassia M.; Eisenberg, Iris; Marshall, Jamie L.; Gasperini, Molly J.; Lek, Angela; Myers, Jennifer A.; Estrella, Elicia A.; Kang, Peter B.; Shapiro, Frederic; Rahimov, Fedik; Kawahara, Genri; Widrick, Jeffrey J.; Kunkel, Louis M. Share Save
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Dysferlin, Annexin A1, and Mitsugumin 53 Are Upregulated in Muscular Dystrophy and Localize to Longitudinal Tubules of the T-System With Stretch Waddell, Leigh B.; Lemckert, Frances A.; Zheng, Xi F.; Tran, Jenny; Evesson, Frances J.; Hawkes, Joanne M.; Lek, Angela; Street, Neil E.; Lin, Peihui; Clarke, Nigel F.; Landstrom, Andrew P.; Ackerman, Michael J.; Weisleder, Noah; Ma, Jianjie; North, Kathryn N.; Cooper, Sandra T. Share Save
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