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

المؤلفون: S. E. Hamm, C. Yuan, L. F. McQueen, M. A. Wallace, H. Zhang, A. Arora, A. M. Garafalo, R. P. McMillan, M. W. Lawlor, M. J. Prom, E. M. Ott, J. Yan, A. K. Addington, C. A. Morris, J. P. Gonzalez, R. W. Grange

المصدر: Frontiers in Physiology, Vol 14 (2023)

مصطلحات موضوعية: endurance, muscle strength, AAV (adeno-associated virus), microdystrophin, longevity, Physiology, QP1-981

وصف الملف: electronic resource

Relation: https://www.frontiersin.org/articles/10.3389/fphys.2023.1166206/full; https://doaj.org/toc/1664-042X

URL الوصول: https://doaj.org/article/0e80ab2a4ff242cab8e283779799e335

المؤلفون: Hiromi Hayashita-Kinoh, Posadas-Herrera Guillermo, Yuko Nitahara-Kasahara, Mutsuki Kuraoka, Hironori Okada, Tomoko Chiyo, Shin’ichi Takeda, Takashi Okada

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 133-141 (2021)

مصطلحات موضوعية: AAV, DMD, immune modulation, MSC, DMD dog model, microdystrophin, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120302308; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/931c70404534486fa01bab2533ae4c67

المؤلفون: Akiko Ishii, Hironori Okada, Hiromi Hayashita-Kinoh, Jin-Hong Shin, Akira Tamaoka, Takashi Okada, Shin’ichi Takeda

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 44-49 (2020)

مصطلحات موضوعية: tacrolimus, muscular dystrophy, recombinant adeno-associated virus, gene therapy, microdystrophin, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120300991; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/a58c999022b54f3a9aef2884be3963e7

المؤلفون: Arianna Manini, Elena Abati, Andi Nuredini, Stefania Corti, Giacomo Pietro Comi

المصدر: Frontiers in Neurology, Vol 12 (2022)

مصطلحات موضوعية: Duchenne muscular dystrophy, adeno-associated virus, gene therapy, persistence, dystrophin, microdystrophin, Neurology. Diseases of the nervous system, RC346-429

وصف الملف: electronic resource

Relation: https://www.frontiersin.org/articles/10.3389/fneur.2021.814174/full; https://doaj.org/toc/1664-2295

URL الوصول: https://doaj.org/article/72642f56f89844db982f53efac823500

المؤلفون: Buel D. Rodgers, Yemeserach Bishaw, Denali Kagel, Julian N. Ramos, Joseph W. Maricelli

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 122-132 (2020)

مصطلحات موضوعية: Duchenne muscular dystrophy, microdystrophin, gene therapy, exercise, mdx, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050119301408; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/40f52319db4042c0855c6b3404dcfa19

المؤلفون: Ho, Peggy P., Lahey, Lauren J., Mourkioti, Foteini, Kraft, Peggy E., Filareto, Antonio, Brandt, Moritz, Magnusson, Klas E. G., Finn, Eric E., Chamberlain, Jeffrey S., Robinson, William H., Blau, Helen M., Steinman, Lawrence

المصدر: Proceedings of the National Academy of Sciences of the United States of America, 2018 Sep . 115(39), E9182-E9191.

URL الوصول: https://www.jstor.org/stable/26531790

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المؤلفون: Nathalie Bourg, Ai Vu Hong, William Lostal, Abbass Jaber, Nicolas Guerchet, Guillaume Tanniou, Fanny Bordier, Emilie Bertil-Froidevaux, Christophe Georger, Nathalie Daniele, Isabelle Richard, David Israeli

المصدر: International Journal of Molecular Sciences, Vol 23, Iss 4, p 2016 (2022)

مصطلحات موضوعية: Duchenne muscular dystrophy, simvastatin, gene therapy, AAV, microdystrophin, combined therapy, Biology (General), QH301-705.5, Chemistry, QD1-999

وصف الملف: electronic resource

Relation: https://www.mdpi.com/1422-0067/23/4/2016; https://doaj.org/toc/1661-6596; https://doaj.org/toc/1422-0067

URL الوصول: https://doaj.org/article/b33d5a168eaa421083eccfe1b5d5b3d0

المؤلفون: Tabebordbar, Mohammadsharif^Aff5, Cheng, Jason^Aff5, Wagers, Amy J.^Aff5

المساهمون: Christen, Yves, Editor^Aff1, Jaenisch, Rudolf, editor^Aff2, Zhang, Feng, editor^Aff3, Gage, Fred, editor^Aff4

المصدر: Genome Editing in Neurosciences. :103-123