[HTML][HTML] Translational regulation of utrophin by miRNAs

U Basu, O Lozynska, C Moorwood, G Patel, SD Wilton… - PloS one, 2011 - journals.plos.org
Background Utrophin is the autosomal homolog of dystrophin, the product of the Duchenne
Muscular Dystrophy (DMD) locus. Its regulation is of therapeutic interest as its …

Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy

DR Chancellor, KE Davies, O De Moor… - Journal of medicinal …, 2011 - ACS Publications
A series of novel 2-arylbenzoxazoles that upregulate the production of utrophin in murine
H2K cells, as assessed using a luciferase reporter linked assay, have been identified. This …

[HTML][HTML] Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping

A Goyenvalle, A Babbs, D Powell, R Kole, S Fletcher… - Molecular Therapy, 2010 - cell.com
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by
mutations in the dystrophin gene that result in the absence of functional protein. Antisense …

Up71 and up140, two novel transcripts of utrophin that are homologues of short forms of dystrophin

J Wilson, W Putt, C Jimenez… - Human Molecular …, 1999 - academic.oup.com
Utrophin is a large protein which accumulates at the neuromuscular synapse and
myotendinous junctions in adult skeletal muscle, and is widely expressed in several non …

[HTML][HTML] Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies

EP Hoffman, EM Connor - Discovery medicine, 2013 - discoverymedicine.com
Duchenne muscular dystrophy is a relatively common'rare disorder,'with an incidence of
about 1/5,000 males worldwide. The responsible gene and deficient protein (dystrophin) …

Distinct regions in the 3′ untranslated region are responsible for targeting and stabilizing utrophin transcripts in skeletal muscle cells

AO Gramolini, G Bélanger, BJ Jasmin - The Journal of cell biology, 2001 - rupress.org
In this study, we have sought to determine whether utrophin transcripts are targeted to a
distinct subcellular compartment in skeletal muscle cells, and have examined the role of the …

Progress in therapy for Duchenne muscular dystrophy

RJ Fairclough, A Bareja, KE Davies - Experimental physiology, 2011 - Wiley Online Library
Duchenne muscular dystrophy is a devastating muscular dystrophy of childhood. Mutations
in the dystrophin gene destroy the link between the internal muscle filaments and the …

The role of basal and myogenic factors in the transcriptional activation of utrophin promoter A: implications for therapeutic up-regulation in Duchenne muscular …

KJ Perkins, EA Burton, KE Davies - Nucleic Acids Research, 2001 - academic.oup.com
Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle wasting disease
caused by the absence of a muscle cytoskeletal protein, dystrophin. Utrophin is the …

Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene

JM Tinsley, AC Potter, SR Phelps, R Fisher, JI Trickett… - Nature, 1996 - nature.com
DUCHENNE muscular dystrophy (DMD) is a severe, progressive muscle-wasting disease
that causes cardiac or respiratory failure1, 2 and results in death at about 20 years of age …

Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles

R Gilbert, J Nalbantoglu, BJ Petrof, S Ebihara… - Human gene …, 1999 - liebertpub.com
Utrophin is a close homolog of dystrophin, the protein whose mutations cause Duchenne
muscular dystrophy (DMD). Utrophin is present at low levels in normal and dystrophic …