Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model von Stoica, Lorelei, Todeasa, Sophia H., Cabrera, Gabriela Toro, Salameh, Johnny S., ElMallah, Mai K., Mueller, Christian, Brown Jr, Robert H., Sena-Esteves, Miguel

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Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing von Gonzalez, Trevor J., Simon, Katherine E., Blondel, Leo O., Fanous, Marco M., Roger, Angela L., Maysonet, Maribel Santiago, Devlin, Garth W., Smith, Timothy J., Oh, Daniel K., Havlik, L. Patrick, Castellanos Rivera, Ruth M., Piedrahita, Jorge A., ElMallah, Mai K., Gersbach, Charles A., Asokan, Aravind

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Pathophysiology of Alpha-1 Antitrypsin Lung Disease von Kalfopoulos, Michael, Wetmore, Kaitlyn, K. ElMallah, Mai

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The respiratory neuromuscular system in Pompe disease von Fuller, David D, ElMallah, Mai K, Smith, Barbara K, Corti, Manuela, Lawson, Lee Ann, Falk, Darin J, Byrne, Barry J

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Macroglossia, Motor Neuron Pathology, and Airway Malacia Contribute to Respiratory Insufficiency in Pompe Disease: A Commentary on Molecular Pathways and Respiratory Involvement in... von McCall, Angela L, ElMallah, Mai K

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Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice von ElMallah, Mai K, Falk, Darin J, Nayak, Sushrusha, Federico, Roland A, Sandhu, Milapjit S, Poirier, Amy, Byrne, Barry J, Fuller, David D

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Respiratory pathology in the TDP-43 transgenic mouse model of amyotrophic lateral sclerosis von Biswas, Debolina D, Sethi, Ronit, Woldeyohannes, Yochebed, Scarrow, Evelyn R, El Haddad, Léa, Lee, Jane, ElMallah, Mai K

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The Respiratory Phenotype of Pompe Disease Mouse Models von Fusco, Anna F, McCall, Angela L, Dhindsa, Justin S, Zheng, Lucy, Bailey, Aidan, Kahn, Amanda F, ElMallah, Mai K

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Airway smooth muscle dysfunction in Pompe ( Gaa -/- ) mice von Keeler, Allison M, Liu, Donghai, Zieger, Marina, Xiong, Lang, Salemi, Jeffrey, Bellvé, Karl, Byrne, Barry J, Fuller, David D, ZhuGe, Ronghua, ElMallah, Mai K

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Neuropathology in respiratory-related motoneurons in young Pompe ( Gaa−/− ) mice von Turner, Sara M.F, Hoyt, Aaron K, ElMallah, Mai K, Falk, Darin J, Byrne, Barry J, Fuller, David D

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The impact of Pompe disease on smooth muscle: a review von MCCALL, Angela L., SALEMI, Jeffrey, BHANAP, Preeti, STRICKLAND, Laura M., ELMALLAH, Mai K.

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Congenital lobar emphysema in monozygotic twins von Scott, Zeni, Zhou, Cynthia, Tracy, Elisabeth, ElMallah, Mai K., Yousef, Shatha

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Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease von Falk, Darin J, Mah, Cathryn S, Soustek, Meghan S, Lee, Kun-Ze, ElMallah, Mai K, Cloutier, Denise A, Fuller, David D, Byrne, Barry J

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Glycogen accumulation in smooth muscle of a Pompe disease mouse model von McCall, Angela L., Dhindsa, Justin S., Bailey, Aidan M., Pucci, Logan A., Strickland, Laura M., ElMallah, Mai K.

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Respiratory dysfunction in a mouse model of spinocerebellar ataxia type 7 von Fusco, Anna F, Pucci, Logan A, Switonski, Pawel M, Biswas, Debolina D, McCall, Angela L, Kahn, Amanda F, Dhindsa, Justin S, Strickland, Laura M, La Spada, Albert R, ElMallah, Mai K

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X-linked SBMA model mice display relevant non-neurological phenotypes and their expression of mutant androgen receptor protein in motor neurons is not required for neuromuscular di... von Gromova, Anastasia, Cha, Byeonggu, Robinson, Erica M, Strickland, Laura M, Nguyen, Nhat, ElMallah, Mai K, Cortes, Constanza J, La Spada, Albert R

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Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model von Keeler, Allison M., Zieger, Marina, Semple, Carson, Pucci, Logan, Veinbachs, Alessandra, Brown, Robert H., Mueller, Christian, ElMallah, Mai K.

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The severity of MUSK pathogenic variants is predicted by the protein domain they disrupt von Cocanougher, Benjamin T., Liu, Samuel W., Francescatto, Ludmila, Behura, Alexander, Anneling, Mariele, Jackson, David G., Deak, Kristen L., Hornik, Chi D., ElMallah, Mai K., Pizoli, Carolyn E., Smith, Edward C., Tan, Khoon Ghee Queenie, McDonald, Marie T.

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Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease von Han, Sang-oh, Li, Songtao, McCall, Angela, Arnson, Benjamin, Everitt, Jeffrey I., Zhang, Haoyue, Young, Sarah P., ElMallah, Mai K., Koeberl, Dwight D.

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GAA Deficiency Disrupts Distal Airway Cells in Pompe Disease von El Haddad, Léa, Lai, Elias, Murthy, Preetish, Tata, Purushothama R., ElMallah, Mai K.

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