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New therapeutic insight into duchenne muscular dystrophy
April 02, 2007In the April 1st issue of Genes & Development, Dr. Bruce Spiegelman (Dana Farber Cancer Institute) and colleagues identify a key genetic component of and possible therapeutic target for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy, affecting about 1 in 3000 males each year. It is an X-linked recessive disease, in which mutations in the dystrophin gene causes progressive and degenerative muscle weakness. DMD is generally lethal by age 30.
Dr. Spiegelman and colleagues found that a protein called PGC-1alpha regulates the point of connection between the end of a motor neuron and a muscle fiber - what researchers call the "neuromuscular junction." Electrical impulses travel through the neuromuscular junction, ultimately causing the muscle to contract. Previous research has shown that PGC-1alpha expression is induced by physical exercise and motor neuron activity, and mediates the anti-atrophic effects of nerve activity on muscle mass.
Dr. Spiegelman and colleagues analyzed the function of PGC-1alpha in a mouse model of DMD. They found that PGC-1alpha activates the expression of several genes that are aberrantly inactivated in DMD. In fact, by inducing PGC-1alpha expression in these transgenic mice, the scientists were able to improve DMD symptoms.
"These data clearly show that experimental elevation of PGC-1 alpha has therapeutic promise in an animal model of Duchene's muscular dystrophy. We hope this will lead eventually to therapeutics for a terrible disease for which there is no effective treatment at the present time," explains Dr. Spiegelman.
Cold Spring Harbor Laboratory
Dr. Spiegelman and colleagues analyzed the function of PGC-1alpha in a mouse model of DMD. They found that PGC-1alpha activates the expression of several genes that are aberrantly inactivated in DMD. In fact, by inducing PGC-1alpha expression in these transgenic mice, the scientists were able to improve DMD symptoms.
"These data clearly show that experimental elevation of PGC-1 alpha has therapeutic promise in an animal model of Duchene's muscular dystrophy. We hope this will lead eventually to therapeutics for a terrible disease for which there is no effective treatment at the present time," explains Dr. Spiegelman.
Cold Spring Harbor Laboratory
Muscular Dystrophy Current Events and Muscular Dystrophy News RSSTreatment to improve degenerating muscle gains strength
A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders.
Possible help in fight against muscle-wasting disease
A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease, according to researchers at the University of Oregon and the University of Rochester School of Medicine and Dentistry in New York.
Exon-skipping drug prevents muscle wasting, maintains muscle function in dystrophin deficient mice
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.
To regenerate muscle, cellular garbage men must become builders
For scientists at the European Molecular Biology Laboratory (EMBL) in Monterotondo, Italy, what seemed like a disappointing result turned out to be an important discovery.
Zoo volunteers help explain mysteries of the genome
As the University of Leicester approaches the 25th anniversary of the discovery of DNA fingerprinting (September 10), Leicester geneticists interested in a particular type of DNA are receiving some help from an unusual band of assistants.
Small molecule inhibits pathology associated with myotonic dystrophy type 1
Researchers at the University of Illinois have designed a small molecule that blocks an aberrant pathway associated with myotonic dystrophy type 1, the most common form of muscular dystrophy.
Researchers identify new function for protein missing in Duchenne muscular dystrophy
Researchers at the University of Minnesota and National Institutes of Health have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy.
Sticky protein helps reinforce fragile muscle membranes
A new study by scientists at the University of Iowa shows why muscle membranes don't rupture when healthy people exercise.
Stem cell surprise for tissue regeneration
Scientists working at the Carnegie Institution's Department of Embryology, with colleagues, have overturned previous research that identified critical genes for making muscle stem cells.
Researchers make progress toward early identification of muscular dystrophy
The saying "Knowing is half the battle" is never more true than when discussing early treatment of disease. Muscular dystrophy is one such disease where patients can benefit from early treatment. Now, new research is moving doctors and scientists closer to disease diagnosis in advance of patient symptoms.
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