...Andrea J. Arreguin; Zijian Shao; Holly Colognato ABSTRACT Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, resulting in the loss of dystrophin, a large cytosolic protein that links the cytoskeleton to extracellular matrix receptors in skeletal muscle. Aside from progressive...

... for duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues . Nucleic Acid Ther. 27 , 251 - 259 . 10.1089/nat.2017.0682 Annexstad , E. J. , Lund-Petersen , I. and Rasmussen , M. ( 2014 ). Duchenne muscular dystrophy . Tidsskr. Nor. Laegeforen...

...Dominic E. Fullenkamp; Alexander B. Willis; Jodi L. Curtin; Ansel P. Amaral; Kyle T. Dittloff; Sloane I. Harris; Ivana A. Chychula; Cory W. Holgren; Paul W. Burridge; Brenda Russell; Alexis R. Demonbreun; Elizabeth M. McNally ABSTRACT Heart failure contributes to Duchenne muscular dystrophy (DMD...

...Dominique O. Riddell; John C. W. Hildyard; Rachel C. M. Harron; Frances Taylor-Brown; Joe N. Kornegay; Dominic J. Wells; Richard J. Piercy ABSTRACT Duchenne muscular dystrophy (DMD), caused by mutations in the dystrophin ( DMD ) gene, is associated with fatal muscle degeneration and atrophy...

... of liver kinase B1 (LKB1) in the altered activation of the master metabolic and epigenetic regulator adenosine monophosphate-activated protein kinase (AMPK) in Duchenne muscular dystrophy has not been investigated so far. Hence, we analyzed both gene and protein levels of LKB1 and its related targets...

...Dominique O. Riddell; John C. W. Hildyard; Rachel C. M. Harron; Natasha L. Hornby; Dominic J. Wells; Richard J. Piercy ABSTRACT Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease, caused by mutations in the dystrophin gene, characterised by cycles of muscle degeneration...

...Abbe H. Crawford; John C. W. Hildyard; Sophie A. M. Rushing; Dominic J. Wells; Maria Diez-Leon; Richard J. Piercy ABSTRACT Duchenne muscular dystrophy (DMD), a fatal musculoskeletal disease, is associated with neurodevelopmental disorders and cognitive impairment caused by brain dystrophin...

...Chady H. Hakim; Hsiao T. Yang; Matthew J. Burke; James Teixeira; Gregory J. Jenkins; N. Nora Yang; Gang Yao; Dongsheng Duan ABSTRACT Aged dystrophin-null canines are excellent models for studying experimental therapies for Duchenne muscular dystrophy, a lethal muscle disease caused by dystrophin...

... ABSTRACT Large-animal models for Duchenne muscular dystrophy (DMD) are crucial for the evaluation of diagnostic procedures and treatment strategies. Pigs cloned from male cells lacking DMD exon 52 ( DMD Δ52) exhibit molecular, clinical and pathological hallmarks of DMD, but die before sexual maturity...

...Amel Saoudi; Faouzi Zarrouki; Catherine Sebrié; Charlotte Izabelle; Aurélie Goyenvalle; Cyrille Vaillend ABSTRACT The exon-52-deleted mdx52 mouse is a critical model of Duchenne muscular dystrophy (DMD), as it features a deletion in a hotspot region of the DMD gene, frequently mutated in patients...

... of Duchenne muscular dystrophy (DMD). Its phenotype is, however, mild, and other mouse models have been explored. The mdx:Cmah −/− mouse carries a human-like mutation in the Cmah gene and has a severe muscle phenotype, but its growth and bone development are unknown. In this study, we compared male mdx...

...Trace L. Stay; Lauren N. Miterko; Marife Arancillo; Tao Lin; Roy V. Sillitoe ABSTRACT Duchenne muscular dystrophy (DMD) is a debilitating and ultimately lethal disease involving progressive muscle degeneration and neurological dysfunction. DMD is caused by mutations in the dystrophin gene, which...

...Dominic J. Wells ABSTRACT Duchenne muscular dystrophy (DMD) is a progressive, fatal, X-linked monogenic muscle disorder caused by mutations in the DMD gene. In order to test treatments for DMD, a range of natural and engineered animal models have been developed, including mice, rats, dogs and pigs...

...Bo Lin; Yang Li; Lu Han; Aaron D. Kaplan; Ying Ao; Spandan Kalra; Glenna C. L. Bett; Randall L. Rasmusson; Chris Denning; Lei Yang ABSTRACT Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene ( DMD ), and is characterized by progressive weakness in skeletal and cardiac...

...Joe W. McGreevy; Chady H. Hakim; Mark A. McIntosh; Dongsheng Duan Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace...