Opportunities for mitochondrial disease gene therapy von Viscomi, Carlo, van den Ameele, Jelle, Meyer, Kathrin C., Chinnery, Patrick F.

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Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart Failure-ANVIAS Study von Sierra-Delgado, Julieth A, Likhite, Shibi, Bautista, Paula K, Gómez-Ochoa, Sergio A, Echeverría, Luis E, Guío, Elizabeth, Vargas, Clara, Serrano, Norma C, Meyer, Kathrin C, Rincon, Melvin Y

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Cross-cultural adaptation, reliability, and validity of the German version of the Pain Catastrophizing Scale von Meyer, Kathrin, Sprott, Haiko, Mannion, Anne Frances

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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates von Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, Jerry R, Burghes, Arthur H M, Kaspar, Brian K

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Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS von Meyer, Kathrin, Ferraiuolo, Laura, Miranda, Carlos J., Likhite, Shibi, McElroy, Sohyun, Renusch, Samantha, Ditsworth, Dara, Lagier-Tourenne, Clotilde, Smith, Richard A., Ravits, John, Burghes, Arthur H., Shaw, Pamela J., Cleveland, Don W., Kolb, Stephen J., Kaspar, Brian K.

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Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism von Ferraiuolo, Laura, Meyer, Kathrin, Sherwood, Thomas W., Vick, Jonathan, Likhite, Shibi, Frakes, Ashley, Miranda, Carlos J., Braun, Lyndsey, Heath, Paul R., Pineda, Ricardo, Beattie, Christine E., Shaw, Pamela J., Askwith, Candice C., McTigue, Dana, Kaspar, Brian K.

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RNA helicase IGHMBP2 regulates THO complex to ensure cellular mRNA homeostasis von Prusty, Archana Bairavasundaram, Hirmer, Anja, Sierra-Delgado, Julieth Andrea, Huber, Hannes, Guenther, Ulf-Peter, Schlosser, Andreas, Dybkov, Olexandr, Yildirim, Ezgi, Urlaub, Henning, Meyer, Kathrin C., Jablonka, Sibylle, Erhard, Florian, Fischer, Utz

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Astrocytes from familial and sporadic ALS patients are toxic to motor neurons von Kaspar, Brian K, Haidet-Phillips, Amanda M, Hester, Mark E, Miranda, Carlos J, Meyer, Kathrin, Braun, Lyndsey, Frakes, Ashley, Song, SungWon, Likhite, Shibi, Murtha, Matthew J, Foust, Kevin D, Rao, Meghan, Eagle, Amy, Kammesheidt, Anja, Christensen, Ashley, Mendell, Jerry R, Burghes, Arthur H M

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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS von Foust, Kevin D, Salazar, Desirée L, Likhite, Shibi, Ferraiuolo, Laura, Ditsworth, Dara, Ilieva, Hristelina, Meyer, Kathrin, Schmelzer, Leah, Braun, Lyndsey, Cleveland, Don W, Kaspar, Brian K

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Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis von Song, SungWon, Miranda, Carlos J, Braun, Lyndsey, Meyer, Kathrin, Frakes, Ashley E, Ferraiuolo, Laura, Likhite, Shibi, Bevan, Adam K, Foust, Kevin D, McConnell, Michael J, Walker, Christopher M, Kaspar, Brian K

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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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Nuclear Factor I in neurons, glia and during the formation of Müller glia‐derived progenitor cells in avian, porcine and primate retinas von El‐Hodiri, Heithem M., Campbell, Warren A., Kelly, Lisa E., Hawthorn, Evan C., Schwartz, Maura, Jalligampala, Archana, McCall, Maureen A., Meyer, Kathrin, Fischer, Andy J.

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SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits von Hautbergue, Guillaume M., Castelli, Lydia M., Ferraiuolo, Laura, Sanchez-Martinez, Alvaro, Cooper-Knock, Johnathan, Higginbottom, Adrian, Lin, Ya-Hui, Bauer, Claudia S., Dodd, Jennifer E., Myszczynska, Monika A., Alam, Sarah M., Garneret, Pierre, Chandran, Jayanth S., Karyka, Evangelia, Stopford, Matthew J., Smith, Emma F., Kirby, Janine, Meyer, Kathrin, Kaspar, Brian K., Isaacs, Adrian M., El-Khamisy, Sherif F., De Vos, Kurt J., Ning, Ke, Azzouz, Mimoun, Whitworth, Alexander J., Shaw, Pamela J.

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Follistatin-induced muscle hypertrophy in aged mice improves neuromuscular junction innervation and function von Iyer, Chitra C., Chugh, Deepti, Bobbili, Prameela J., III, Anton J. Blatnik, Crum, Alexander E., Yi, Allen F., Kaspar, Brian K., Meyer, Kathrin C., Burghes, Arthur H.M., Arnold, W. David

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AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease von Johnson, Tyler B., White, Katherine A., Brudvig, Jon J., Cain, Jacob T., Langin, Logan, Pratt, Melissa A., Booth, Clarissa D., Timm, Derek J., Davis, Samantha S., Meyerink, Brandon, Likhite, Shibi, Meyer, Kathrin, Weimer, Jill M.

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MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndrome von Dennys, Cassandra N., Vermudez, Sheryl Anne D., Deacon, Robert J.M., Sierra-Delgado, J. Andrea, Rich, Kelly, Zhang, Xiaojin, Buch, Aditi, Weiss, Kelly, Moxley, Yuta, Rajpal, Hemangi, Espinoza, Francisca D., Powers, Samantha, Ávila, Ariel S., Gogliotti, Rocco G., Cogram, Patricia, Niswender, Colleen M., Meyer, Kathrin C.

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The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome von Jeon, Shin, Park, Jaein, Likhite, Shibi, Moon, Ji Hwan, Shin, Dongjun, Li, Liwen, Meyer, Kathrin C., Lee, Jae W., Lee, Soo-Kyung

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Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy von Sinha Ray, Shrestha, Dutta, Debdeep, Dennys, Cassandra, Powers, Samantha, Roussel, Florence, Lisowski, Pawel, Glažar, Petar, Zhang, Xiaojin, Biswas, Pipasha, Caporale, Joseph R., Rajewsky, Nikolaus, Bickle, Marc, Wein, Nicolas, Bellen, Hugo J., Likhite, Shibi, Marcogliese, Paul C., Meyer, Kathrin C.

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Good Attitudes Are Not Good Enough: An Ethnographical Approach to Investigate Attitude-Behavior Inconsistencies in Sustainable Choice von Meyer, Kathrin Barbara, Simons, Johannes

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AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models von Murakami, Yoshiko, Umeshita, Saori, Imanishi, Kae, Yoshioka, Yoshichika, Ninomiya, Akinori, Sunabori, Takehiko, Likhite, Shibi, Koike, Masato, Meyer, Kathrin C., Kinoshita, Taroh

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