dystrophy

Muscle-targeted gene therapy reverses rare muscular dystrophy in mice

Gene therapy methods that specifically target muscle may reverse the symptoms of a rare form of muscular dystrophy, according to new research in mice conducted by medical geneticists at Duke University Medical Center. Infants born with the inherited muscular disorder called Pompe disease usually die before they reach the age of two. The researchers also said their approach of targeting corrective...

Chemical 'band-aid' prevents heart failure in mice with muscular dystrophy

A common chemical used in the manufacturing and pharmaceutical industries can repair damage to cardiac muscle cell membranes and prevent heart failure in mice with the genetic mutation that causes Duchenne muscular dystrophy, according to scientists at the University of Michigan Medical School. The mutation in the dystrophin gene causes the progressive deterioration of skeletal muscles see...

No small feat: First ever gene therapy success for muscular dystrophy achieved

Researchers from the University of Pittsburgh report the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable scientific challenges that have cast doubt on gene therapy ever being feasible for children with muscular dystrophy can be overcome. Moreover, their results, published in this week's online edi...

Gene therapy reverses genetic mutation responsible for heart failure in muscular dystrophy

University of Pittsburgh investigators have for the first time used gene therapy to successfully treat heart failure and other degenerative muscle problems in an animal model that is genetically susceptible to a human muscular dystrophy. Reporting in the Oct. 25 edition of the journal Circulation, the authors say that this is the first successful attempt to deliver a therapeutic gene throughout t...

Gene therapy for muscular dystrophy fixes frail muscle cells in animal model, Stanford study finds

A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, according to a new study by researchers at Stanford University School of Medicine. In the study, scheduled to be published online in the Proceedings of the National Academy of Sciences the week of Jan. 2, the researchers used gene therapy to introduce...

Toxic molecule may cause most common type of muscular dystrophy

Doctors at the University of Virginia Health System have shown for the first time that getting rid of poisonous RNA (ribonucleic acid) in muscle cells can reverse myotonic dystrophy, the most common type of muscular dystrophy in adults. About 40,000 people in the United States have myotonic muscular dystrophy (MMD). The disease can cause a slow, progressive wasting of the muscles, irregul...

Experimental cancer drugs counter muscle deterioration seen in muscular dystrophy

Muscle weakness and fiber deterioration seen in muscular dystrophy can be countered by a class of drugs currently under study for their effects against cancer, a Burnham Institute study has found. The report shed light on the potential use of these drugs, called histone deacetylase inhibitors, in promoting regeneration and repair of dystrophic muscles, thereby countering the progression o...

Scientists show drug can counteract muscular dystrophy in mice

Scientists at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other institutions have demonstrated for the first time that a single drug can rebuild damaged muscle in two strains of mice that develop diseases comparable to two human forms of muscular dystrophy. This advance, which is reported online in Nature Medicine, is the latest from a research collaborat...

Switching genes to overdrive improves muscular dystrophy symptoms in mice

Scientists at Dana-Farber Cancer Institute have shown in a laboratory study that revving up a crucial set of muscle genes counteracts the damage caused by a form of muscular dystrophy. Reporting in the April 1 issue of Genes and Development, the researchers demonstrated that manipulating a genetic molecular switch increased the genes?activity in the muscles of mice with Duchenne muscular d...