P.315Src tyrosine kinase as a drug target in Duchenne muscular dystrophy: a proof-of-concept study in the exercised mdx mouse

2019 ◽  
Vol 29 ◽  
pp. S159
Author(s):  
F. Sanarica ◽  
B. Boccanegra ◽  
P. Mantuano ◽  
O. Cappellari ◽  
M. De Bellis ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Tyrosine Kinase ◽  
Drug Target ◽  
Mdx Mouse ◽  
Proof Of Concept ◽  
Concept Study

scholarly journals Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study

PLoS ONE ◽  
2016 ◽  
Vol 11 (1) ◽  
pp. e0147634 ◽  
Author(s):  
Patricia Hafner ◽  
Ulrike Bonati ◽  
Beat Erne ◽  
Maurice Schmid ◽  
Daniela Rubino ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Function ◽  
Proof Of Concept ◽  
Single Center ◽  
Open Label ◽  
Concept Study

scholarly journals Functional and Molecular Effects of Arginine Butyrate and Prednisone on Muscle and Heart in the mdx Mouse Model of Duchenne Muscular Dystrophy

PLoS ONE ◽  
2010 ◽  
Vol 5 (6) ◽  
pp. e11220 ◽  
Author(s):  
Alfredo D. Guerron ◽  
Rashmi Rawat ◽  
Arpana Sali ◽  
Christopher F. Spurney ◽  
Emidio Pistilli ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Mdx Mouse ◽  
Molecular Effects ◽  
Arginine Butyrate

scholarly journals Macrophages escape Klotho gene silencing in the mdx mouse model of Duchenne muscular dystrophy and promote muscle growth and increase satellite cell numbers through a Klotho-mediated pathway

2017 ◽  
Vol 27 (1) ◽  
pp. 14-29 ◽  
Author(s):  
Michelle Wehling-Henricks ◽  
Steven S Welc ◽  
Guiseppina Samengo ◽  
Chiara Rinaldi ◽  
Catherine Lindsey ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Gene Silencing ◽  
Satellite Cell ◽  
Muscle Growth ◽  
Mdx Mouse ◽  
Cell Numbers ◽  
Klotho Gene

scholarly journals Correction: Functional and Molecular Effects of Arginine Butyrate and Prednisone on Muscle and Heart in the mdx Mouse Model of Duchenne Muscular Dystrophy

PLoS ONE ◽  
2010 ◽  
Vol 5 (8) ◽  
Author(s):  
Alfredo D. Guerron ◽  
Rashmi Rawat ◽  
Arpana Sali ◽  
Christopher F. Spurney ◽  
Emidio Pistilli ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Mdx Mouse ◽  
Molecular Effects ◽  
Arginine Butyrate

scholarly journals Metabolomic Analyses Reveal Extensive Progenitor Cell Deficiencies in a Mouse Model of Duchenne Muscular Dystrophy

Metabolites ◽  
2018 ◽  
Vol 8 (4) ◽  
pp. 61 ◽  
Author(s):  
Josiane Joseph ◽  
Dong Cho ◽  
Jason Doles
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Progenitor Cell ◽  
Treatment Options ◽  
Cell Types ◽  
Mdx Mouse ◽  
Mdx Mice ◽  
Metabolic Alterations ◽  
Potential Contributor

Duchenne muscular dystrophy (DMD) is a musculoskeletal disorder that causes severe morbidity and reduced lifespan. Individuals with DMD have an X-linked mutation that impairs their ability to produce functional dystrophin protein in muscle. No cure exists for this disease and the few therapies that are available do not dramatically delay disease progression. Thus, there is a need to better understand the mechanisms underlying DMD which may ultimately lead to improved treatment options. The muscular dystrophy (MDX) mouse model is frequently used to explore DMD disease traits. Though some studies of metabolism in dystrophic mice exist, few have characterized metabolic profiles of supporting cells in the diseased environment. Using nontargeted metabolomics we characterized metabolic alterations in muscle satellite cells (SCs) and serum of MDX mice. Additionally, live-cell imaging revealed MDX-derived adipose progenitor cell (APC) defects. Finally, metabolomic studies revealed a striking elevation of acylcarnitines in MDX APCs, which we show can inhibit APC proliferation. Together, these studies highlight widespread metabolic alterations in multiple progenitor cell types and serum from MDX mice and implicate dystrophy-associated metabolite imbalances in APCs as a potential contributor to adipose tissue disequilibrium in DMD.

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