Добірка наукової літератури з теми "Microdystrophin"
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Статті в журналах з теми "Microdystrophin"
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.
Повний текст джерелаBrown, 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.
Повний текст джерелаHersh, 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.
Повний текст джерелаHo, 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.
Повний текст джерелаMartin, 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.
Повний текст джерелаBostick, 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.
Повний текст джерелаPercival, 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.
Повний текст джерелаBoehler, 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.
Повний текст джерелаShin, Jin-Hong, Xiufang Pan, Chady H. Hakim, Hsiao T. Yang, Yongping Yue, Keqing Zhang, Ronald L. Terjung, and Dongsheng Duan. "Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy." Molecular Therapy 21, no. 4 (April 2013): 750–57. http://dx.doi.org/10.1038/mt.2012.283.
Повний текст джерелаPichavant, Christophe, Pierre Chapdelaine, Daniel G. Cerri, Jean-Christophe Dominique, Simon P. Quenneville, Daniel Skuk, Joe N. Kornegay, João CS Bizario, Xiao Xiao, and Jacques P. Tremblay. "Expression of Dog Microdystrophin in Mouse and Dog Muscles by Gene Therapy." Molecular Therapy 18, no. 5 (May 2010): 1002–9. http://dx.doi.org/10.1038/mt.2010.23.
Повний текст джерелаДисертації з теми "Microdystrophin"
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.
Повний текст джерелаDuchenne 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
Частини книг з теми "Microdystrophin"
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.
Повний текст джерелаFoster, 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.
Повний текст джерела