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Articles tagged with duchenne muscular dystrophy

Inhibition of TGF-beta

The transforming growth factor beta (TGFβ) is a protein which inhibits the satellite cells (muscle stem cells) from regenerating muscle tissue. Mdx mice and also Duchenne boys have increa...

Tags: Duchenne Muscular Dystrophy, fibrosis, mdx mice, pirfenidone, TGF946.

MEDICAL ISSUES- PART 1

For a child with Duchenne the primary school years are usually a time of significant change in terms of their independent mobility. When particular changes occur is a little variable but the patte...

Tags: Duchenne muscular dystrophy, MOBILITY, WEAKness in the thighs, physical activies.

2-'O-methyl antisense oligos (PS) targeting exon 44.

BackgroundAntisense-mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allo...

Tags: antisense oligonucleotides, Duchenne muscular dystrophy, exon skipping, 28242Omethyl phosphorothioate, phosphorodiamidate morpholino oligomer.

By-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skipping.

BackgroundDuchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by protein-truncating mutations in the dystrophin gene. Absence of functional dystrophin renders muscle fibr...

Tags: antisense oligomer, B6RosCgDmdmdx4CvJ 4CV mouse, Duchenne muscular dystrophy, exon skipping, morpholino.

An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in duchenne-like muscular dystrophy in the corgi breed

Duchenne muscular dystrophy (DMD) is a dystrophin-deficient lethal muscle disease. To date, the catastrophic muscle wasting phenotype has only been seen in dystrophin-deficient human and dogs. Alt...

Tags: dystrophin, Duchenne muscular dystrophy, DMD.

Targeting Fibrosis in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease affecting 1 in 3,500 live male births. It is an X-linked recessive disease caused by a defective dystrophin gene. The di...

Tags: Duchenne muscular dystrophy, Fibrosis, muscle, cell therapies.

Interleukin-10 reduces the pathology of mdx muscular dystrophy by deactivating M1 macrophages and modulating macrophage phenotype.

Molecular, Cellular & Integrative Physiology Program, University of California, Los Angeles, CA.M1 macrophages play a major role in worsening muscle injury in the mdx mouse model of Duchenne m...

Tags: mdx muscles, dystrophy, Duchenne muscular dystrophy.

Duchenne muscular dystrophy - What causes the increased membrane permeability in skeletal muscle?

Duchenne muscular dystrophy is a severe muscle wasting disease caused by a mutation in the gene for dystrophin – a cytoskeletal protein connecting the contractile machinery to a group of pro...

Tags: muscle weakness, atrophy, therapies, Duchenne muscular dystrophy.

GSK2402968 Clinical Development Programme Update

Forty-Eight Week Follow-Up Data from a Phase I/IIa Extension Study of PRO051/GSK2402968 in Subjects with Duchenne Muscular DystrophyThe following Phase I/IIa extension study results were presented...

Tags: Duchenne, DMD, GSK, Duchenne Muscular Dystrophy.

£2.5m research grant for Duchenne Muscular Dystrophy awarded to Oxford researchers

Funding for pioneering research into gene therapy by Action Duchenne has led to further funds for research into this life limiting disease A recent £2.5 million award made by the Health Innovation...

Tags: exon skipping, Duchenne, Duchenne muscular dystrophy, Dr Matthew Wood, Mike Gait, antisense oligonucleotides.