Results 71 to 80 of about 7,866 (244)

Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.

PLoS ONE, 2011
BackgroundDuchenne muscular dystrophy (DMD) is a lethal, progressive muscle wasting disease caused by a loss of sarcolemmal bound dystrophin, which results in the death of the muscle fibers leading to the gradual depletion of skeletal muscle.
Jonathon M Tinsley, Rebecca J Fairclough, Richard Storer, Fraser J Wilkes, Allyson C Potter, Sarah E Squire, Dave S Powell, Anna Cozzoli, Roberta F Capogrosso, Adam Lambert, Francis X Wilson, Stephen P Wren, Annamaria De Luca, Kay E Davies   +13 more
doaj   +1 more source

Alternative Splicing: Molecular Mechanisms, Biological Functions, Diseases, and Potential Therapeutic Targets

MedComm, Volume 6, Issue 12, December 2025.
Alternative splicing (AS) expands proteomic diversity and functional complexity in eukaryotes, regulated by spliceosomal components, RNA elements, and epigenetic modifications. Dysregulated AS contributes to diseases, including cancer, neurodegenerative disorders, and cardiovascular conditions, among others. Therapeutic interventions, such as antisense
Zhi‐Min Zhu, Xiao‐Mei Wu, Yan Hu, Xiao‐Lan Bian, Ya‐Qin Wang, Qiong‐Ni Zhu   +5 more
wiley   +1 more source

Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects.

PLoS Genetics, 2020
Gene therapy approaches for DMD using recombinant adeno-associated viral (rAAV) vectors to deliver miniaturized (or micro) dystrophin genes to striated muscles have shown significant progress.
Glen B Banks, Jeffrey S Chamberlain, Guy L Odom   +2 more
doaj   +1 more source

Actin behavior in bulk cytoplasm is cell cycle regulated in early vertebrate embryos [PDF]

, 2011
The mechanical properties of cells change as they proceed through the cell cycle, primarily owing to regulation of actin and myosin II. Most models for cell mechanics focus on actomyosin in the cortex and ignore possible roles in bulk cytoplasm.
Abe, Agnew, Amano, Belmont, Bendix, Boucrot, Bray, Brieher, Burkel, Chang, Clark, Condeelis, Cytrynbaum, Dao, Desai, Ding, Eggert, Elinson, Ezzell, Gibson, Hara, Kaji, Kane, Kueh, Kunda, Lenart, Loisel, Maddox, Martin, Matsumura, May, Mitchison, Murray, Murray, Niethammer, Pollard, Pollard, Rankin, Riedl, Rosenblatt, Rosenblatt, Schroeder, Taunton, Theriot, Theriot, Valentine, Velarde, Verde, Waterman-Storer, Welch, Woolner, Wühr, Wühr   +52 more
core   +3 more sources

Exploring Desmin as a Potential Modifier in Duchenne Muscular Dystrophy–Associated Cardiomyopathy

Acta Physiologica, Volume 241, Issue 12, December 2025.
ABSTRACT Aim Duchenne muscular dystrophy (DMD), a rare X‐linked genetic disorder, is affecting skeletal and cardiac muscles due to the loss of the dystrophin protein. Modifier proteins, whose expression is altered in DMD patients, may influence disease progression.
Brice‐Emmanuel Guennec, Yeranuhi Hovhannisyan, Gaëlle Revet, Sila Polat, Medhi Hassani, Nathalie Mougenot, Inès Barthelemy, Stephane Blot, Caroline Cieniewski‐Bernard, Arnaud Ferry, Ekaterini Kordeli, Zhenlin Li, Onnik Agbulut   +12 more
wiley   +1 more source

Functional substitution by TAT-utrophin in dystrophin-deficient mice. [PDF]

PLoS Medicine, 2009
The loss of dystrophin compromises muscle cell membrane stability and causes Duchenne muscular dystrophy and/or various forms of cardiomyopathy. Increased expression of the dystrophin homolog utrophin by gene delivery or pharmacologic up-regulation has ...
Kevin J Sonnemann, Hanke Heun-Johnson, Amy J Turner, Kristen A Baltgalvis, Dawn A Lowe, James M Ervasti   +5 more
doaj   +1 more source

Aryl Hydrocarbon Receptor in Health and Disease

MedComm, Volume 6, Issue 11, November 2025.
Based on the structure and ligands of AhR, this review introduces the AhR‐related signaling pathways and their roles in health and diseases. Agonists and antagonists of AhR as well as new strategies for treatment using the microbial–AhR axis are summarized. A prospect was made for the future use of AhR as a therapeutic target.
Haonan Li, Yufeng Fan, Jizheng Liu, Shumin Dong, Bin Wen, Yunfei Zhang, Xiaocui Wang, Xuemei Duan, Ying Hu, Ze Yan, Huifeng Shang, Yukai Jing   +11 more
wiley   +1 more source

Estrogen‐Related Receptor Alpha Promotes Skeletal Muscle Regeneration and Mitigates Muscular Dystrophy

The FASEB Journal, Volume 39, Issue 19, 15 October 2025.
ERRα promotes muscle regeneration. ERRα drives angiogenic and mitochondrial metabolic gene program in proliferating and differentiating myogenic cells. ERRα also induces myogenic factor genes such as MyoG. Through these pathways ERRα promotes muscle regeneration in the skeletal muscle in acute injury and chronic myopathy.
Thi Thu Hao Nguyen, Ya Xiang Huang, Svitlana Poliakova, Citu Citu, Eira Mann, Danesh H. Sopariwala, Zhongming Zhao, Ashok Kumar, Vihang A. Narkar   +8 more
wiley   +1 more source

Endogenous bioluminescent reporters reveal a sustained increase in utrophin gene expression upon EZH2 and ERK1/2 inhibition

Communications Biology, 2023
With the long-term goal of finding therapeutic options for Duchenne muscular dystrophy, a mouse model enabling simultaneous visualisation of Dmd and Utrn is described and inhibitors of PRC2 and ERK1/2 identified to increase utrophin expression.
Hannah J. Gleneadie, Beatriz Fernandez-Ruiz, Alessandro Sardini, Mathew Van de Pette, Andrew Dimond, Rab K. Prinjha, James McGinty, Paul M. W. French, Hakan Bagci, Matthias Merkenschlager, Amanda G. Fisher   +10 more
doaj   +1 more source

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene [PDF]

Gene Therapy, 2000
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a subsarcolemmal protein, dystrophin. We have previously shown that the dystrophin-related protein, utrophin is able to compensate for the lack of dystrophin in the mdx mouse, the mouse model for DMD.
Wakefield, P. M., Tinsley, J. M., Wood, M. J. A., Gilbert, R., Karpati, G., Davies, K. E.   +5 more
openaire   +4 more sources