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1دورية أكاديمية
المؤلفون: Muntoni, FrancescoAff1, Aff2, Byrne, Barry J., McMillan, Hugh J., Ryan, Monique M., Wong, Brenda L., Dukart, JuergenAff7, Aff8, Bansal, Amita, Cosson, Valerie, Dreghici, RoxanaAff9, Aff10, Guridi, Maitea, Rabbia, Michael, Staunton, Hannah, Tirucherai, Giridhar S., Yen, KarlAff11, Aff14, Yuan, Xiling, Wagner, Kathryn R.Aff9, Aff15, IDs4012002300570w_cor16
المصدر: Neurology and Therapy. 13(1):183-219
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2دورية أكاديمية
المؤلفون: Chamberlain, Jeffrey S, Robb, Melissa, Braun, Serge, Brown, Kristy J, Danos, Olivier, Ganot, Annie, Gonzalez-Alegre, Pedro, Hunter, Nina, McDonald, Craig, Morris, Carl, Tobolowsky, Mark, Wagner, Kathryn R, Ziolkowski, Olivia, Duan, Dongsheng
المصدر: Human Gene Therapy. 34(9-10)
مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Regenerative Medicine, Clinical Trials and Supportive Activities, Biotechnology, Intellectual and Developmental Disabilities (IDD), Gene Therapy, Orphan Drug, Duchenne/ Becker Muscular Dystrophy, Pediatric, Brain Disorders, Muscular Dystrophy, Rare Diseases, Genetics, Clinical Research, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Musculoskeletal, Male, Humans, Muscular Dystrophy, Duchenne, Muscle, Skeletal, Genetic Therapy, Gene Transfer Techniques, Biomarkers, microdystrophin, Duchenne muscular dystrophy, gene therapy, AAV vector, Clinical Sciences, Medical biotechnology
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/0v56c0vk
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3دورية أكاديمية
المؤلفون: Chiriboga, Claudia A.Aff1, IDs40120023005037_cor1, Bruno, Claudio, Duong, Tina, Fischer, Dirk, Mercuri, Eugenio, Kirschner, Janbernd, Kostera-Pruszczyk, AnnaAff7, Aff8, Jaber, Birgit, Gorni, Ksenija, Kletzl, Heidemarie, Carruthers, Imogen, Martin, Carmen, Warren, Francis, Scalco, Renata S., Wagner, Kathryn R., Muntoni, Francesco
المصدر: Neurology and Therapy. 12(5):1799-1801
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4دورية أكاديمية
المؤلفون: Tawil, Rabi *, Wagner, Kathryn R *, Hamel, Johanna I, Leung, Doris G, Statland, Jeffrey M, Wang, Leo H, Genge, Angela, Sacconi, Sabrina, Lochmüller, Hanns, Reyes-Leiva, David, Diaz-Manera, Jordi, Alonso-Perez, Jorge, Muelas, Nuria, Vilchez, Juan J, Pestronk, Alan, Gibson, Summer, Goyal, Namita A, Hayward, Lawrence J, Johnson, Nicholas, LoRusso, Samantha, Freimer, Miriam, Shieh, Perry B, Subramony, S H, van Engelen, Baziel, Kools, Joost, Leinhard, Olof Dahlqvist, Widholm, Per, Morabito, Christopher, Moxham, Christopher M, Cadavid, Diego, Mellion, Michelle L, Odueyungbo, Adefowope, Tracewell, William G, Accorsi, Anthony, Ronco, Lucienne, Gould, Robert J, Shoskes, Jennifer, Rojas, Luis Alejandro, Jiang, John G *
المصدر: In The Lancet Neurology May 2024 23(5):477-486
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5دورية أكاديمية
المؤلفون: Britson, Kyla A, Ling, Jonathan P, Braunstein, Kerstin E, Montagne, Janelle M, Kastenschmidt, Jenna M, Wilson, Andrew, Ikenaga, Chiseko, Tsao, William, Pinal-Fernandez, Iago, Russell, Katelyn A, Reed, Nicole, Mozaffar, Tahseen, Wagner, Kathryn R, Ostrow, Lyle W, Corse, Andrea M, Mammen, Andrew L, Villalta, S Armando, Larman, H Benjamin, Wong, Philip C, Lloyd, Thomas E
المصدر: Science translational medicine. 14(628)
مصطلحات موضوعية: Muscle, Skeletal, CD8-Positive T-Lymphocytes, Vacuoles, Animals, Humans, Mice, Myositis, Myositis, Inclusion Body, DNA-Binding Proteins, Heterografts, Clinical Research, Neurosciences, Genetics, Biotechnology, Neurodegenerative, Aetiology, 2.1 Biological and endogenous factors, Neurological, Biological Sciences, Medical and Health Sciences
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/00p558pw
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6دورية أكاديمية
المؤلفون: Chiriboga, Claudia A.Aff1, IDs40120023004441_cor1, Bruno, Claudio, Duong, Tina, Fischer, Dirk, Mercuri, Eugenio, Kirschner, Janbernd, Kostera-Pruszczyk, AnnaAff7, Aff8, Jaber, Birgit, Gorni, Ksenija, Kletzl, Heidemarie, Carruthers, Imogen, Martin, Carmen, Warren, Francis, Scalco, Renata S., Wagner, Kathryn R., Muntoni, Francesco
المصدر: Neurology and Therapy. 12(2):543-557
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7دورية أكاديمية
المؤلفون: Rutten-Jacobs, Loes, McIver, Tammy, Reyes, Adriana, Pereira, Marta, Rosenthal, Rachel, Parusel, Christine T., Wagner, Kathryn R., Doody, Rachelle
المصدر: In Contemporary Clinical Trials Communications February 2024 37
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8دورية أكاديمية
المؤلفون: Wang, Leo H., Leung, Doris G., Wagner, Kathryn R., Lowry, Sarah J., McDermott, Michael P., Eichinger, Katy, Higgs, Kiley, Walker, Michaela, Lewis, Leann, Martens, William B., Mul, Karlien, Sansone, Valeria A., Shieh, Perry, Elsheikh, Bakri, LoRusso, Samantha, Butterfield, Russell J., Johnson, Nicholas, Preston, Matthew R., Messina, Carmelo, Carraro, Elena, Tawil, Rabi, Statland, Jeff
المصدر: In Neuromuscular Disorders September 2023 33(9):63-68
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9دورية أكاديمية
المؤلفون: Sherlock, Sarah P.Aff1, IDs00415022110840_cor1, Palmer, Jeffrey, Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Bertini, Enrico, Tian, CuixiaAff5, Aff6, Mah, Jean K., Kostera-Pruszczyk, Anna, Muntoni, Francesco, Guglieri, Michela, Brandsema, John F., Mercuri, EugenioAff12, Aff13, Butterfield, Russell J., McDonald, Craig M., Charnas, Lawrence, Marraffino, Shannon
المصدر: Journal of Neurology. 269(8):4421-4435
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10دورية أكاديمية
المؤلفون: Sherlock, Sarah P.Aff1, IDs41598022230725_cor1, Palmer, Jeffrey, Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Tian, CuixiaAff4, Aff5, Mah, Jean K., Muntoni, Francesco, Guglieri, Michela, Butterfield, Russell J., Charnas, Lawrence, Marraffino, Shannon
المصدر: Scientific Reports. 12(1)