[HTML][HTML] Translational regulation of utrophin by miRNAs
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 …
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 …
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 …
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 …
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) …
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 …
distinct subcellular compartment in skeletal muscle cells, and have examined the role of the …
Progress in therapy for Duchenne muscular dystrophy
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 …
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 …
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 …
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 …
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 …
muscular dystrophy (DMD). Utrophin is present at low levels in normal and dystrophic …