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https://www.readbyqxmd.com/read/29121992/a-mouse-anti-myostatin-antibody-increases-muscle-mass-and-improves-muscle-strength-and-contractility-in-the-mdx-mouse-model-of-duchenne-muscular-dystrophy-and-its-humanized-equivalent-domagrozumab-pf-06252616-increases-muscle-volume-in-cynomolgus-monkeys
#1
Michael St Andre, Mark Johnson, Prashant N Bansal, Jeremy Wellen, Andrew Robertson, Alan Opsahl, Peter M Burch, Peter Bialek, Carl Morris, Jane Owens
BACKGROUND: The treatments currently approved for Duchenne muscular dystrophy (DMD), a progressive skeletal muscle wasting disease, address the needs of only a small proportion of patients resulting in an urgent need for therapies that benefit all patients regardless of the underlying mutation. Myostatin is a member of the transforming growth factor-β (TGF-β) family of ligands and is a negative regulator of skeletal muscle mass. Loss of myostatin has been shown to increase muscle mass and improve muscle function in both normal and dystrophic mice...
November 9, 2017: Skeletal Muscle
https://www.readbyqxmd.com/read/29103911/cyclic-peptides-to-improve-delivery-and-exon-skipping-of-antisense-oligonucleotides-in-a-mouse-model-for-duchenne-muscular-dystrophy
#2
Silvana M G Jirka, Peter A C 't Hoen, Valeriano Diaz Parillas, Christa L Tanganyika-de Winter, Ruurd C Verheul, Begona Aguilera, Peter C de Visser, Annemieke M Aartsma-Rus
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder caused by reading frame disrupting mutations in the DMD gene. Exon skipping is a therapeutic approach for DMD. It employs antisense oligonucleotides (AONs) to restore the disrupted open reading frame, allowing the production of shorter, but partly functional dystrophin protein as seen in less severely affected Becker muscular dystrophy patients. To be effective, AONs need to be delivered and effectively taken up by the target cells, which can be accomplished by the conjugation of tissue-homing peptides...
October 12, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29095865/beneficial-effects-of-high-dose-taurine-treatment-in-juvenile-dystrophic-mdx-mice-are-offset-by-growth-restriction
#3
Jessica R Terrill, Gavin J Pinniger, Keshav V Nair, Miranda D Grounds, Peter G Arthur
Duchenne Muscular Dystrophy (DMD) is a fatal muscle wasting disease manifested in young boys, for which there is no current cure. We have shown that the amino acid taurine is safe and effective at preventing dystropathology in the mdx mouse model for DMD. This study aimed to establish if treating growing mdx mice with a higher dose of taurine was more effective at improving strength and reducing inflammation and oxidative stress. Mice were treated with a dose of taurine estimated to be 16 g/kg/day, in drinking water from 1-6 weeks of age, after which in vivo and ex vivo muscle strength was assessed, as were measures of inflammation, oxidative stress and taurine metabolism...
2017: PloS One
https://www.readbyqxmd.com/read/29072171/effects-of-dietary-omega-3-on-dystrophic-cardiac-and-diaphragm-muscles-as-evaluated-by-1-h-magnetic-resonance-spectroscopy-metabolic-profile-and-calcium-related-proteins
#4
Adriana Fogagnolo Maurício, Samara Camaçari de Carvalho, Humberto Santo Neto, Maria Julia Marques
BACKGROUND & AIMS: Duchenne muscular dystrophy (DMD) is characterized by the absence of dystrophin and muscle degeneration. Calcium dysregulation and oxidative stress also contribute to the disease progression. We evaluated the potential therapeutic benefits of supplementation with omega-3 on the metabolic profile, calcium-related proteins and oxidative stress response in the heart and diaphragm (DIA) of the mdx mouse model of DMD at later stages of the disease (13 months). METHODS: Mdx mice (8 months old) received omega-3 via a dietary supplement for 5 months...
August 2017: Clinical Nutrition ESPEN
https://www.readbyqxmd.com/read/29067664/pmo-delivery-system-using-bubble-liposomes-and-ultrasound-exposure-for-duchenne-muscular-dystrophy-treatment
#5
Yoichi Negishi, Yuko Ishii, Kei Nirasawa, Eri Sasaki, Yoko Endo-Takahashi, Ryo Suzuki, Kazuo Maruyama
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration, caused by nonsense or frameshift mutations in the dystrophin (DMD) gene. Antisense oligonucleotides can be used to induce specific exon skipping; recently, a phosphorodiamidate morpholino oligomer (PMO) has been approved for clinical use in DMD. However, an efficient PMO delivery strategy is required to improve the therapeutic efficacy in DMD patients. We previously developed polyethylene glycol (PEG)-modified liposomes containing ultrasound contrast gas, "Bubble liposomes" (BLs), and found that the combination of BLs with ultrasound exposure is a useful gene delivery tool...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29067663/the-use-of-antisense-oligonucleotides-for-the-treatment-of-duchenne-muscular-dystrophy
#6
Karima Relizani, Aurelie Goyenvalle
Antisense oligonucleotides (AONs) hold great promise for therapeutic splice-switching correction in many genetic diseases and in particular for Duchenne muscular dystrophy (DMD), where AONs can be used to reframe the dystrophin transcript and give rise to a partially deleted but yet functional dystrophin protein. Many different chemistries of AONs can be used for splice switching modulation, and some of them have been evaluated in clinical trials for DMD. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake, and sufficient therapeutic efficacy is difficult to achieve...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29067660/exon-skipping-therapy-using-phosphorodiamidate-morpholino-oligomers-in-the-mdx52-mouse-model-of-duchenne-muscular-dystrophy
#7
Shouta Miyatake, Yoshitaka Mizobe, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Shin'ichi Takeda, Yoshitsugu Aoki
Exon skipping therapy using synthetic DNA-like molecules called antisense oligonucleotides (ASOs) is a promising therapeutic candidate for overcoming the dystrophin mutation that causes Duchenne muscular dystrophy (DMD). This treatment involves splicing out the frame-disrupting segment of the dystrophin mRNA, which restores the reading frame and produces a truncated yet functional dystrophin protein. Phosphorodiamidate morpholino oligomer (PMO) is the safest ASO for patients among ASOs and has recently been approved under the accelerated approval pathway by the U...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29067659/probing-the-pathogenesis-of-duchenne-muscular-dystrophy-using-mouse-models
#8
Alexander Morrison-Nozik, Saptarsi M Haldar
Investigations using mouse models have provided seminal insights into the pathogenesis of Duchenne muscular dystrophy and the development of novel therapeutics. Several important methods have been considered standard-in-the-field for analyses of skeletal muscle weakness, strength, endurance, and histopathology, as well as responses to therapeutics such as glucocorticoids, disease modifying drugs which are part of the current standard of care for patients with this disease. Here we describe optimized genetic, genomic, and physiologic assays to probe dystrophic pathobiology in the mdx mouse and related strains...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29067656/imaging-analysis-of-the-neuromuscular-junction-in-dystrophic-muscle
#9
Stephen J P Pratt, Shama R Iyer, Sameer B Shah, Richard M Lovering
Duchenne muscular dystrophy (DMD), caused by the absence of the protein dystrophin, is characterized as a neuromuscular disease in which muscle weakness, increased susceptibility to muscle injury, and inadequate repair appear to underlie the pathology. Considerable attention has been dedicated to studying muscle fiber damage, but there is little information to determine if damage from contraction-induced injury also occurs at or near the nerve terminal axon. Interestingly, both human patients and the mouse model for DMD (the mdx mouse) present fragmented neuromuscular junction (NMJ) morphology...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29067655/characterization-of-the-inflammatory-response-in-dystrophic-muscle-using-flow-cytometry
#10
Jenna M Kastenschmidt, Ileen Avetyan, S Armando Villalta
Although mutations of the dystrophin gene are the causative defect in Duchenne muscular dystrophy (DMD) patients, secondary disease processes such as inflammation contribute greatly to the pathogenesis of DMD. Genetic and histological studies have shown that distinct facets of the immune system promote muscle degeneration or regeneration during muscular dystrophy through mechanisms that are only beginning to be defined. Although histological methods have allowed the enumeration and localization of immune cells within dystrophic muscle, they are limited in their ability to assess the full spectrum of phenotypic states of an immune cell population and its functional characteristics...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29051551/reducing-sarcolipin-expression-mitigates-duchenne-muscular-dystrophy-and-associated-cardiomyopathy-in-mice
#11
Antanina Voit, Vishwendra Patel, Ronald Pachon, Vikas Shah, Mohammad Bakhutma, Erik Kohlbrenner, Joseph J McArdle, Louis J Dell'Italia, Jerry R Mendell, Lai-Hua Xie, Roger J Hajjar, Dongsheng Duan, Diego Fraidenraich, Gopal J Babu
Sarcolipin (SLN) is an inhibitor of the sarco/endoplasmic reticulum (SR) Ca(2+) ATPase (SERCA) and is abnormally elevated in the muscle of Duchenne muscular dystrophy (DMD) patients and animal models. Here we show that reducing SLN levels ameliorates dystrophic pathology in the severe dystrophin/utrophin double mutant (mdx:utr (-/-)) mouse model of DMD. Germline inactivation of one allele of the SLN gene normalizes SLN expression, restores SERCA function, mitigates skeletal muscle and cardiac pathology, improves muscle regeneration, and extends the lifespan...
October 20, 2017: Nature Communications
https://www.readbyqxmd.com/read/29045431/functional-improvement-of-dystrophic-muscle-by-repression-of-utrophin-let-7c-interaction
#12
Manoj K Mishra, Emanuele Loro, Kasturi Sengupta, Steve D Wilton, Tejvir S Khurana
Duchenne muscular dystrophy (DMD) is a fatal genetic disease caused by an absence of the 427kD muscle-specific dystrophin isoform. Utrophin is the autosomal homolog of dystrophin and when overexpressed, can compensate for the absence of dystrophin and rescue the dystrophic phenotype of the mdx mouse model of DMD. Utrophin is subject to miRNA mediated repression by several miRNAs including let-7c. Inhibition of utrophin: let-7c interaction is predicted to 'repress the repression' and increase utrophin expression...
2017: PloS One
https://www.readbyqxmd.com/read/29040534/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
#13
Michelle Wehling-Henricks, Steven S Welc, Guiseppina Samengo, Chiara Rinaldi, Catherine Lindsey, Ying Wang, Jeongyoon Lee, Makoto Kuro-O, James G Tidball
Duchenne muscular dystrophy (DMD) is a muscle wasting disease in which inflammation influences the severity of pathology. We found that the onset of muscle inflammation in the mdx mouse model of DMD coincides with large increases in expression of pro-inflammatory cytokines [tumor necrosis factor-α (TNFα); interferon gamma (IFNγ)] and dramatic reductions of the pro-myogenic protein Klotho in muscle cells and large increases of Klotho in pro-regenerative, CD206+ macrophages. Furthermore, TNFα and IFNγ treatments reduced Klotho in muscle cells and increased Klotho in macrophages...
October 12, 2017: Human Molecular Genetics
https://www.readbyqxmd.com/read/28989703/an-amphipathic-trans-acting-phosphorothioate-rna-element-delivers-an-uncharged-phosphorodiamidate-morpholino-sequence-in-mdx-mouse-myotubes
#14
H V Jain, J F Boehler, D Verthelyi, K Nagaraju, S L Beaucage
An efficient method for the delivery of uncharged polyA-tailed phosphorodiamidate morpholino sequences (PMO) in mammalian cells consists of employing a synthetic 8-mer amphipathic trans-acting poly-2'-O-methyluridylic thiophosphate triester element (2'-OMeUtaPS) as a transfection reagent. Unlike the dTtaPS DNA-based element, this RNA element is potent at delivering polyA-tailed PMO sequences to HeLa pLuc 705 cells or to myotube muscle cells. However, much like dTtaPS, the 2'-OMeUtaPS-mediated internalization of PMO sequences occurs through an energy-dependent mechanism; macropinocytosis appears to be the predominant endocytic pathway used for cellular uptake...
2017: RSC Advances
https://www.readbyqxmd.com/read/28966053/proteome-analysis-in-dystrophic-mdx-mouse-muscle-reveals-a-drastic-alteration-of-key-metabolic-and-contractile-proteins-after-chronic-exercise-and-the-potential-modulation-by-anti-oxidant-compounds
#15
Tania Gamberi, Tania Fiaschi, Elisa Valocchia, Alessandra Modesti, Paola Mantuano, Jean-Francois Rolland, Francesca Sanarica, Annamaria De Luca, Francesca Magherini
Weakness and fatigability are typical features of Duchenne muscular dystrophy patients and are aggravated in dystrophic mdx mice by chronic treadmill exercise. In the present study, we describe, the pattern of differentially abundant spots that is associated to the worsening of dystrophy phenotype induced by chronic exercise. Our proteomic analysis pointed out 34 protein spots with different abundance between sedentary and exercised mdx mice. These proteins belong mostly to glucose metabolism, energy production and sarcomere structure categories...
January 6, 2018: Journal of Proteomics
https://www.readbyqxmd.com/read/28952155/sensorimotor-control-of-breathing-in-the-mdx-mouse-model-of-duchenne-muscular-dystrophy
#16
David P Burns, Arijit Roy, Eric F Lucking, Fiona B McDonald, Sam Gray, Richard J Wilson, Deirdre Edge, Ken D O'Halloran
KEY POINTS: Respiratory failure is a leading cause of mortality in Duchenne muscular dystrophy (DMD), but little is known about the control of breathing in DMD and animal models. We show that young (8 weeks of age) mdx mice hypoventilate during basal breathing due to reduced tidal volume. Basal CO2 production is equivalent in wild-type and mdx mice. We show that carotid bodies from mdx mice have blunted responses to hyperoxia, revealing hypoactivity in normoxia. However, carotid body, ventilatory and metabolic responses to hypoxia are equivalent in wild-type and mdx mice...
November 1, 2017: Journal of Physiology
https://www.readbyqxmd.com/read/28932757/a-five-repeat-micro-dystrophin-gene-ameliorated-dystrophic-phenotype-in-the-severe-dba-2j-mdx-model-of-duchenne-muscular-dystrophy
#17
Chady H Hakim, Nalinda B Wasala, Xiufang Pan, Kasun Kodippili, Yongping Yue, Keqing Zhang, Gang Yao, Brittney Haffner, Sean X Duan, Julian Ramos, Joel S Schneider, N Nora Yang, Jeffrey S Chamberlain, Dongsheng Duan
Micro-dystrophins are highly promising candidates for treating Duchenne muscular dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust disease rescue in the severe DBA/2J-mdx model with a neuronal nitric oxide synthase (nNOS)-binding micro-dystrophin vector. 2 × 10(13) vector genome particles/mouse of the vector were delivered intravenously to 10-week-old mice and were evaluated at 6 months of age. Saturated micro-dystrophin expression was detected in all skeletal muscles and the heart and restored the dystrophin-associated glycoprotein complex and nNOS...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28918017/efficacy-and-safety-profile-of-tricyclo-dna-antisense-oligonucleotides-in-duchenne-muscular-dystrophy-mouse-model
#18
Karima Relizani, Graziella Griffith, Lucía Echevarría, Faouzi Zarrouki, Patricia Facchinetti, Cyrille Vaillend, Christian Leumann, Luis Garcia, Aurélie Goyenvalle
Antisense oligonucleotides (AONs) hold promise for therapeutic splice-switching correction in many genetic diseases. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake and sufficient therapeutic efficacy is still difficult to achieve. A novel class of AONs made of tricyclo-DNA (tcDNA) is considered very promising for the treatment of Duchenne muscular dystrophy (DMD), a neuromuscular disease typically caused by frameshifting deletions or nonsense mutations in the gene-encoding dystrophin and characterized by progressive muscle weakness, cardiomyopathy, and respiratory failure in addition to cognitive impairment...
September 15, 2017: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/28899419/increased-plasma-lipid-levels-exacerbate-muscle-pathology-in-the-mdx-mouse-model-of-duchenne-muscular-dystrophy
#19
Nadia Milad, Zoe White, Arash Y Tehrani, Stephanie Sellers, Fabio M V Rossi, Pascal Bernatchez
BACKGROUND: Duchenne muscular dystrophy (DMD) is caused by loss of dystrophin expression and leads to severe ambulatory and cardiac function decline. However, the dystrophin-deficient mdx murine model of DMD only develops a very mild form of the disease. Our group and others have shown vascular abnormalities in animal models of MD, a likely consequence of the fact that blood vessels express the same dystrophin-associated glycoprotein complex (DGC) proteins as skeletal muscles. METHODS: To test the blood vessel contribution to muscle damage in DMD, mdx (4cv) mice were given elevated lipid levels via apolipoprotein E (ApoE) gene knockout combined with normal chow or lipid-rich Western diets...
September 12, 2017: Skeletal Muscle
https://www.readbyqxmd.com/read/28887840/pre-clinical-evaluation-of-n-acetylcysteine-reveals-side-effects-in-the-mdx-mouse-model-of-duchenne-muscular-dystrophy
#20
Gavin J Pinniger, Jessica R Terrill, Evanna B Assan, Miranda D Grounds, Peter G Arthur
KEY POINTS: Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disease associated with increased inflammation and oxidative stress. The antioxidant N-acetylcysteine (NAC) has been proposed as a therapeutic intervention for DMD boys, but potential adverse effects of NAC have not been widely investigated. We used young (6 weeks old) growing mdx mice to investigate the capacity of NAC supplementation (2% in drinking water for 6 weeks) to improve dystrophic muscle function and to explore broader systemic effects of NAC treatment...
September 9, 2017: Journal of Physiology
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