Academic literature on the topic 'Microdystrophine'
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Journal articles on the topic "Microdystrophine"
Cernisova, Viktorija, Ngoc Lu-Nguyen, Jessica Trundle, Shan Herath, Alberto Malerba, and Linda Popplewell. "Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy." International Journal of Molecular Sciences 24, no. 9 (May 3, 2023): 8174. http://dx.doi.org/10.3390/ijms24098174.
Full textDickson, G., C. Le Guiner, M. Montus, L. Servais, Y. Cherel, J. Y. Hogrel, P. Carlier, et al. "T.I.3 Perspectives on microdystrophins and delivery." Neuromuscular Disorders 23, no. 9-10 (October 2013): 847. http://dx.doi.org/10.1016/j.nmd.2013.06.719.
Full textChamberlain, J., J. Ramos, K. Hollinger, J. Crudele, N. Bengtsson, and S. Hauschka. "Development of microdystrophins for gene therapy of DMD." Neuromuscular Disorders 27 (October 2017): S245. http://dx.doi.org/10.1016/j.nmd.2017.06.539.
Full textHersh, Jessica, José Manuel Condor Capcha, Camila Iansen Irion, Guerline Lambert, Mauricio Noguera, Mohit Singh, Avinash Kaur, et al. "Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery." Pharmaceutics 13, no. 12 (December 15, 2021): 2159. http://dx.doi.org/10.3390/pharmaceutics13122159.
Full textHo, Peggy P., Lauren J. Lahey, Foteini Mourkioti, Peggy E. Kraft, Antonio Filareto, Moritz Brandt, Klas E. G. Magnusson, et al. "Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy." Proceedings of the National Academy of Sciences 115, no. 39 (September 4, 2018): E9182—E9191. http://dx.doi.org/10.1073/pnas.1808648115.
Full textBrown, K., M. Lawlor, D. Golebiowski, P. Gonzalez, V. Ricotti, J. Schneider, and C. Morris. "Quantification of microdystrophin and correlation to circulating biomarkers." Neuromuscular Disorders 27 (October 2017): S214. http://dx.doi.org/10.1016/j.nmd.2017.06.431.
Full textMartin, Paul T., Rui Xu, Louise R. Rodino-Klapac, Elaine Oglesbay, Marybeth Camboni, Chrystal L. Montgomery, Kim Shontz, et al. "Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice." American Journal of Physiology-Cell Physiology 296, no. 3 (March 2009): C476—C488. http://dx.doi.org/10.1152/ajpcell.00456.2008.
Full textBostick, Brian, Jin-Hong Shin, Yongping Yue, and Dongsheng Duan. "AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice." Molecular Therapy 19, no. 10 (October 2011): 1826–32. http://dx.doi.org/10.1038/mt.2011.154.
Full textPercival, Justin M., Paul Gregorevic, Guy L. Odom, Glen B. Banks, Jeffrey S. Chamberlain, and Stanley C. Froehner. "rAAV6-Microdystrophin Rescues Aberrant Golgi Complex Organization in mdx Skeletal Muscles." Traffic 8, no. 10 (July 12, 2007): 1424–39. http://dx.doi.org/10.1111/j.1600-0854.2007.00622.x.
Full textBoehler, Jessica F., Valeria Ricotti, J. Patrick Gonzalez, Meghan Soustek-Kramer, Lauren Such, Kristy J. Brown, Joel S. Schneider, and Carl A. Morris. "Membrane recruitment of nNOSµ in microdystrophin gene transfer to enhance durability." Neuromuscular Disorders 29, no. 10 (October 2019): 735–41. http://dx.doi.org/10.1016/j.nmd.2019.08.009.
Full textDissertations / Theses on the topic "Microdystrophine"
Jaber, Abbass. "Lysosomal defects in Duchenne muscular dystrophy : advancing combined therapeutic approaches." Electronic Thesis or Diss., université Paris-Saclay, 2024. http://www.theses.fr/2024UPASL055.
Full textDuchenne Muscular Dystrophy (DMD) is a muscle degenerative disease primarily affecting young boys, characterized by the loss of dystrophin expression. While gene therapy targeting the restoration of a functional truncated form of dystrophin, known as µ-dystrophin, has shown promise in preclinical studies, its therapeutic efficacy in treated DMD patients remains limited, necessitating urgent improvements. The aim of the work presented in this thesis is to first improve our understanding of the metabolic and cellular perturbations in DMD, and secondly to propose new combined therapy approaches, associating gene therapy to treatments of identified dysregulations. We have recently identified dysregulations of cholesterol metabolism in DMD muscle, a phenomenon frequently associated with lysosomal dysfunction in neurodegenerative disorders. Building upon this association, we hypothesized a potential interplay between cholesterol accumulation and lysosomal perturbations in DMD pathogenesis. Our study identified an upregulation of Galectin-3 (LGALS3), a known biomarker of lysosome membrane permeabilization (LMP), in the dystrophic muscle of DMD patients and animal models, indicating the occurrence of LMP within dystrophic myofibers. This correlated with significant lysosomal stress evidenced by changes in lysosome number, morphology, positioning and activation of lysosomal biogenesis, repair and removal pathways. Remarkably, LMP was exacerbated in mice fed a cholesterol-rich diet and was not fully corrected by µ-dystrophin gene therapy. Subsequently, we selected trehalose, an FDA-approved compound known to restore lysosomal function, for use in combination with a suboptimal dose of AAV-µ-dystrophin gene therapy. The combined treatment resulted in correction of lysosomal defects and improved correction of dystrophic parameters, including motor function, muscle histology, and transcriptome signature, compared to µ-dystrophin gene therapy alone. This work underscores the significance of lysosomal damage in DMD pathophysiology and suggests that a synergistic approach combining trehalose supplementation with a suboptimal dose of AAV µ-dystrophin holds promise for enhancing therapeutic outcomes in DMD
Book chapters on the topic "Microdystrophine"
Athanasopoulos, Takis, Helen Foster, Keith Foster, and George Dickson. "Codon Optimization of the Microdystrophin Gene for Duchenne Muscular Dystrophy Gene Therapy." In Methods in Molecular Biology, 21–37. Totowa, NJ: Humana Press, 2010. http://dx.doi.org/10.1007/978-1-61737-982-6_2.
Full textFoster, Helen, Taeyoung Koo, Alberto Malerba, Susan Jarmin, Takis Athanasopoulos, Keith Foster, and George Dickson. "A1-4 Microdystrophin and myostatin gene therapy for Duchenne muscular dystrophy using adeno-associated virus vectors." In The CliniBook, 46–54. EDP Sciences, 2012. http://dx.doi.org/10.1051/978-2-84254-237-5.c009.
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