Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice [PDF]
Regenerative TherapyBackground: Therapeutic activation of endogenous utrophin, a homolog of dystrophin, represents a mutation-agnostic therapeutic strategy with considerable potential for treating Duchenne muscular dystrophy (DMD).
Zhikai Xiahou +8 more
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Differential requirement for utrophin in the induced pluripotent stem cell correction of muscle versus fat in muscular dystrophy mice. [PDF]
PLoS ONE, 2011 Duchenne muscular dystrophy (DMD) is an incurable degenerative muscle disorder. We injected WT mouse induced pluripotent stem cells (iPSCs) into mdx and mdx∶utrophin mutant blastocysts, which are predisposed to develop DMD with an increasing degree of ...
Amanda J Beck +9 more
doaj +3 more sources
IRES-mediated translation of utrophin A is enhanced by glucocorticoid treatment in skeletal muscle cells. [PDF]
PLoS ONE, 2008 Glucocorticoids are currently the only drug treatment recognized to benefit Duchenne muscular dystrophy (DMD) patients. The nature of the mechanisms underlying the beneficial effects remains incompletely understood but may involve an increase in the ...
Pedro Miura +3 more
doaj +3 more sources
Molecular Metabolism, 2021 Objectives: Preferential damage to fast, glycolytic myofibers is common in many muscle-wasting diseases, including Duchenne muscular dystrophy (DMD).
Justin P. Hardee +16 more
doaj +3 more sources
Activation of endogenous full-length utrophin by MyoAAV-UA as a therapeutic approach for Duchenne muscular dystrophy [PDF]
Nature CommunicationsActivation of endogenous full-length utrophin, a dystrophin homolog, presents an attractive therapeutic strategy for Duchenne muscular dystrophy (DMD), regardless of mutation types and loci.
Ruo Wu +18 more
doaj +2 more sources
L-Arginine Activates the Neuregulin-1/ErbB Receptor Signaling Pathway and Increases Utrophin mRNA Levels in C2C12 Cells [PDF]
Biochemistry Research InternationalL-arginine induces the expression of utrophin in skeletal muscle cells, so it has been proposed as a pharmacological treatment to attenuate the symptoms of Duchenne muscular dystrophy (DMD).
Gladys Tapia +6 more
doaj +2 more sources
Biallelic variants in the UTRN gene cause a novel form of multiple congenital arthrogryposis [PDF]
Frontiers in GeneticsArthrogryposis multiplex congenita (AMC) is a large group of congenital conditions characterized by joint contractures affecting two or more body areas.
Evgeniya Melnik +9 more
doaj +2 more sources
Muscle structure influences utrophin expression in mdx mice. [PDF]
PLoS Genetics, 2014 Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder caused by mutations in the dystrophin gene. To examine the influence of muscle structure on the pathogenesis of DMD we generated mdx4cv:desmin double knockout (dko) mice.
Glen B Banks +4 more
doaj +3 more sources
Utrophin up-regulation helps maintain normal cardiac geometry in a gene therapy model for Duhenne muscular dystrophy heart disease [abstract] [PDF]
, 2010 Duchenne muscular dystrophy (DMD) is the most common childhood muscle wasting disease. DMD sufferers rarely survive past their mid-twenties succumbing to respiratory or heart failure.
Bostick, Brian P., 1979-
core +2 more sources
Respiratory pathology in the mdx/utrn -/- mouse: A murine model for Duchenne Muscular Dystrophy (DMD). [PDF]
PLoS ONEDuchenne muscular dystrophy (DMD) is an X-linked devastating disease caused by a lack of dystrophin which results in progressive muscle weakness. As muscle weakness progresses, respiratory insufficiency and hypoventilation result in significant morbidity
Marán Y Hernández Rodríguez +10 more
doaj +2 more sources