keyword
MENU ▼
Read by QxMD icon Read
search

MDX

keyword
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/29120619/a-rapid-throughput-method-to-extract-dna-from-formalin-fixed-paraffin-embedded-tissues-for-biomonitoring-carcinogenic-dna-adducts-in-humans
#2
Byeong Hwa Yun, Shun Xiao, Lihua Yao, Sesha Krishnamachari, Thomas A Rosenquist, Kathleen G Dickman, Arthur P Grollman, Paari Murugan, Christopher J Weight, Robert J Turesky
Formalin-fixed paraffin-embedded (FFPE) tissues are rarely used for screening DNA adducts of carcinogens because the harsh conditions required to reverse the formaldehyde-mediated DNA cross-links can destroy DNA adducts. We recently adapted a commercial silica-based column kit used in genomics to manually isolate DNA under mild conditions from FFPE tissues of rodents and humans and successfully measured DNA adducts of several carcinogens including aristolochic acid I (AA-I), 4-aminobiphenyl (4-ABP), and 2-amino-1-methyl-6-phenylimidazo[4,5-b]pyridine (PhIP) (Yun et al...
November 9, 2017: Chemical Research in Toxicology
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
#3
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/29097503/ryanodine-channel-complex-stabilizer-compound-s48168-arm210-as-a-disease-modifier-in-dystrophin-deficient-mdx-mice-proof-of-concept-study-and-independent-validation-of-efficacy
#4
Roberta Francesca Capogrosso, Paola Mantuano, Kitipong Uaesoontrachoon, Anna Cozzoli, Arcangela Giustino, Todd Dow, Sadish Srinivassane, Marina Filipovic, Christina Bell, Jack Vandermeulen, Ada Maria Massari, Michela De Bellis, Elena Conte, Sabata Pierno, Giulia Maria Camerino, Antonella Liantonio, Kanneboyina Nagaraju, Annamaria De Luca
Muscle fibers lacking dystrophin undergo a long-term alteration of Ca(2+) homeostasis, partially caused by a leaky Ca(2+) release ryanodine (RyR) channel. S48168/ARM210, an RyR calcium release channel stabilizer (a Rycal compound), is expected to enhance the rebinding of calstabin to the RyR channel complex and possibly alleviate the pathologic Ca(2+) leakage in dystrophin-deficient skeletal and cardiac muscle. This study systematically investigated the effect of S48168/ARM210 on the phenotype of mdx mice by means of a first proof-of-concept, short (4 wk), phase 1 treatment, followed by a 12 wk treatment (phase 2) performed in parallel by 2 independent laboratories...
November 2, 2017: FASEB Journal: Official Publication of the Federation of American Societies for Experimental Biology
https://www.readbyqxmd.com/read/29095865/beneficial-effects-of-high-dose-taurine-treatment-in-juvenile-dystrophic-mdx-mice-are-offset-by-growth-restriction
#5
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/29079520/zac1-gpr39-phosphorylating-camk-ii-contributes-to-the-distinct-roles-of-pax3-and-pax7-in-myogenic-progression
#6
Qiumei Yang, Ye Li, Xulong Zhang, Daiwen Chen
Both Pax3 and Pax7 can activate a large panel of genes involved in muscle stem cell function. Despite a significant overlap in their transcriptional network, functional difference between them is observed. After overexpressing Pax3 or Pax7 in C2C12, we find both Zac1 and GPR39 are upregulated by Pax7 but not Pax3. Further studies suggest Zac1 interacts directly with Pax7, which can regulate GPR39 expression by activating Zac1. In addition, the effect of Zac1/GPR39 system on myogenic progression has been illuminated: Zac1/GPR39 can promote myogenic differentiation and produce type-II muscle fibers...
November 2, 2017: Biochimica et Biophysica Acta
https://www.readbyqxmd.com/read/29078808/treatment-with-the-anti-il-6-receptor-antibody-attenuates-muscular-dystrophy-via-promoting-skeletal-muscle-regeneration-in-dystrophin-utrophin-deficient-mice
#7
Eiji Wada, Jun Tanihata, Akira Iwamura, Shin'ichi Takeda, Yukiko K Hayashi, Ryoichi Matsuda
BACKGROUND: Chronic increases in the levels of the inflammatory cytokine interleukin-6 (IL-6) in serum and skeletal muscle are thought to contribute to the progression of muscular dystrophy. Dystrophin/utrophin double-knockout (dKO) mice develop a more severe and progressive muscular dystrophy than the mdx mice, the most common murine model of Duchenne muscular dystrophy (DMD). In particular, dKO mice have smaller body sizes and muscle diameters, and develop progressive kyphosis and fibrosis in skeletal and cardiac muscles...
October 27, 2017: Skeletal Muscle
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
#8
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
#9
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
#10
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
#11
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
#12
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
#13
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
#14
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/29065908/utrophin-influences-mitochondrial-pathology-and-oxidative-stress-in-dystrophic-muscle
#15
Tahnee L Kennedy, Lee Moir, Sarah Hemming, Ben Edwards, Sarah Squire, Kay Davies, Simon Guiraud
BACKGROUND: Duchenne muscular dystrophy (DMD) is a lethal X-linked muscle wasting disorder caused by the absence of dystrophin, a large cytoskeletal muscle protein. Increasing the levels of the dystrophin-related-protein utrophin is a highly promising therapy for DMD and has been shown to improve pathology in dystrophin-deficient mice. One contributing factor to muscle wasting in DMD is mitochondrial pathology that contributes to oxidative stress and propagates muscle damage. The purpose of this study was to assess whether utrophin could attenuate mitochondria pathology and oxidative stress...
October 24, 2017: Skeletal Muscle
https://www.readbyqxmd.com/read/29051551/reducing-sarcolipin-expression-mitigates-duchenne-muscular-dystrophy-and-associated-cardiomyopathy-in-mice
#16
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
#17
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
#18
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/29036556/deficit-in-pink1-parkin-mediated-mitochondrial-autophagy-at-late-stages-of-dystrophic-cardiomyopathy
#19
Chifei Kang, Myriam A Badr, Viktoriia Kyrychenko, Eeva-Liisa Eskelinen, Natalia Shirokova
Aims: Duchenne Muscular Dystrophy (DMD) is an inherited devastating muscle disease with severe and often lethal cardiac complications. Emerging evidence suggests that the evolution of the pathology in DMD is accompanied by the accumulation of mitochondria with defective structure and function. Here we investigate whether defects in the housekeeping autophagic pathway contribute to mitochondrial and metabolic dysfunctions in dystrophic cardiomyopathy. Methods and results: We employed various biochemical and imaging techniques to assess mitochondrial structure and function as well as to evaluate autophagy, and specific mitochondrial autophagy (mitophagy), in hearts of mdx mice, an animal model of DMD...
October 5, 2017: Cardiovascular Research
https://www.readbyqxmd.com/read/28989703/an-amphipathic-trans-acting-phosphorothioate-rna-element-delivers-an-uncharged-phosphorodiamidate-morpholino-sequence-in-mdx-mouse-myotubes
#20
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
keyword
keyword
3755
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"