Relevant bibliographies by topics / Microdystrophine

Contents

  1. Journal articles
  2. Dissertations / Theses
  3. Book chapters

Academic literature on the topic 'Microdystrophine'

Author: Grafiati
Published: 9 November 2024

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Journal articles on the topic "Microdystrophine"

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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 (2023): 8174. http://dx.doi.org/10.3390/ijms24098174.

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Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease affecting 1:5000 newborn males. No cure is currently available, but gene addition therapy, based on the adeno-associated viral (AAV) vector-mediated delivery of microdystrophin transgenes, is currently being tested in clinical trials. The muscles of DMD boys present significant fibrotic and adipogenic tissue deposition at the time the treatment starts. The presence of fibrosis not only worsens the disease pathology, but also diminishes the efficacy of gene therapy treatments. To gain an understanding of the efficacy of AAV-based
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Dickson, G., C. Le Guiner, M. Montus, et al. "T.I.3 Perspectives on microdystrophins and delivery." Neuromuscular Disorders 23, no. 9-10 (2013): 847. http://dx.doi.org/10.1016/j.nmd.2013.06.719.

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Chamberlain, 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.

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Hersh, Jessica, José Manuel Condor Capcha, Camila Iansen Irion, et al. "Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery." Pharmaceutics 13, no. 12 (2021): 2159. http://dx.doi.org/10.3390/pharmaceutics13122159.

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Gene therapy is a good alternative for determined congenital disorders; however, there are numerous limitations for gene delivery in vivo including targeted cellular uptake, intracellular trafficking, and transport through the nuclear membrane. Here, a modified G5 polyamidoamine (G5 PAMAM) dendrimer–DNA complex was developed, which will allow cell-specific targeting to skeletal muscle cells and transport the DNA through the intracellular machinery and the nuclear membrane. The G5 PAMAM nanocarrier was modified with a skeletal muscle-targeting peptide (SMTP), a DLC8-binding peptide (DBP) for in
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Ho, Peggy P., Lauren J. Lahey, Foteini Mourkioti, 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 (2018): E9182—E9191. http://dx.doi.org/10.1073/pnas.1808648115.

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In gene therapy for Duchenne muscular dystrophy there are two potential immunological obstacles. An individual with Duchenne muscular dystrophy has a genetic mutation in dystrophin, and therefore the wild-type protein is “foreign,” and thus potentially immunogenic. The adeno-associated virus serotype-6 (AAV6) vector for delivery of dystrophin is a viral-derived vector with its own inherent immunogenicity. We have developed a technology where an engineered plasmid DNA is delivered to reduce autoimmunity. We have taken this approach into humans, tolerizing to myelin proteins in multiple sclerosi
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Brown, K., M. Lawlor, D. Golebiowski, et al. "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.

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7

Martin, Paul T., Rui Xu, Louise R. Rodino-Klapac, 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 (2009): C476—C488. http://dx.doi.org/10.1152/ajpcell.00456.2008.

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The cytotoxic T cell (CT) GalNAc transferase, or Galgt2, is a UDP-GalNAc:β1,4- N-acetylgalactosaminyltransferase that is localized to the neuromuscular synapse in adult skeletal muscle, where it creates the synaptic CT carbohydrate antigen {GalNAcβ1,4[NeuAc(orGc)α2, 3]Galβ1,4GlcNAcβ-}. Overexpression of Galgt2 in the skeletal muscles of transgenic mice inhibits the development of muscular dystrophy in mdx mice, a model for Duchenne muscular dystrophy. Here, we provide physiological evidence as to how Galgt2 may inhibit the development of muscle pathology in mdx animals. Both Galgt2 transgenic
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8

Bostick, 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 (2011): 1826–32. http://dx.doi.org/10.1038/mt.2011.154.

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9

Percival, 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 (2007): 1424–39. http://dx.doi.org/10.1111/j.1600-0854.2007.00622.x.

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10

Boehler, Jessica F., Valeria Ricotti, J. Patrick Gonzalez, et al. "Membrane recruitment of nNOSµ in microdystrophin gene transfer to enhance durability." Neuromuscular Disorders 29, no. 10 (2019): 735–41. http://dx.doi.org/10.1016/j.nmd.2019.08.009.

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Dissertations / Theses on the topic "Microdystrophine"

1

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.

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La dystrophie musculaire de Duchenne (DMD) est une maladie musculaire dégénérative touchant principalement les jeunes garçons, caractérisée par la perte de l'expression fonctionnelle de la dystrophine. Bien que la thérapie génique visant à restaurer une forme tronquée fonctionnelle de la dystrophine, appelée µ-dystrophine, ait montré des résultats prometteurs dans les études précliniques, son efficacité thérapeutique chez les patients DMD traités reste limitée, nécessitant des améliorations urgentes. Le travail présenté dans cette thèse vise d'abord à améliorer notre compréhension des perturba
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Book chapters on the topic "Microdystrophine"

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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. Humana Press, 2010. http://dx.doi.org/10.1007/978-1-61737-982-6_2.

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Foster, Helen, Taeyoung Koo, Alberto Malerba, et al. "A1-4 Microdystrophin and myostatin gene therapy for Duchenne muscular dystrophy using adeno-associated virus vectors." In The CliniBook. EDP Sciences, 2012. http://dx.doi.org/10.1051/978-2-84254-237-5.c009.

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