Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
Scientists in the United States say an experimental drug may be able to compensate for the genetic error responsible for some cases of Duchenne Muscular Dystrophy (DMD) and cystic fibrosis.
Female stem cells derived from muscle have a greater ability to regenerate skeletal muscle tissue than male cells, according to a study at Children's Hospital of Pittsburgh of UPMC.
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.
In 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.
Italian scientists, by using new stem cell therapy on dogs with muscular dystrophy, have improved the dogs condition to such an extent that the dogs were able to walk and even jump again.
New approaches to genetic disease, based on cells' own ability to correct themselves, will be outlined today at the annual conference of the European Society of Human Genetics in Amsterdam, The Netherlands.
The clinical trial for Duchenne muscular dystrophy (DMD) tests the safety and effectiveness of a therapy that was developed over two decades by scientists at the University of North Carolina Chapel Hill's School of Medicine and the University of Pittsburgh.
In a surprising discovery with implications for treating muscular dystrophy, researchers at the University of Utah School of Medicine and other institutions have identified a major source of origin for two groups of adult cells that regulate muscle repair.
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.
A new study provides important insight into the mechanisms of a muscle wasting disorder that interferes with treatment for cancer and has a negative impact on patient survival.
New research shows that a wasting condition responsible for nearly a third of all cancer deaths involves the loss of an essential muscle protein that is also lost in people with muscular dystrophy.
Cardiac disease, particularly dilated cardiomyopathy and heart failure, is the major cause of mortality in patients with muscular dystrophy and is present in most boys with Duchenne muscular dystrophy and approximately 70 percent of those with Becker muscular dystrophy. These are the two common forms of muscular dystrophy caused by defects in a gene called dystrophin.
Adult stem cells taken from bone marrow are the "shooting stars" of their field. Many research scientists have been speculating that the cells might be able to pass through the blood into diseased organs and replace defective tissue. Such cells are seen as the potential key to the treatment of certain muscle diseases.
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.
Corticosteroids can be beneficial in the treatment of Duchenne muscular dystrophy and can be offered as a treatment option, according to the American Academy of Neurology and the Child Neurology Society in a new practice guideline published in the January 11 issue of Neurology, the scientific journal of the American Academy of Neurology.
Researchers at the University of Pennsylvania School of Medicine report a novel strategy for stimulating the production of utrophin – an important muscle protein in young mice – for muscular dystrophy therapy.
MDA-supported researchers at the University of Washington-Seattle have delivered the gene for the dystrophin protein to all voluntary muscles with a single intravenous injection in mice with Duchenne muscular dystrophy (DMD).
The Muscular Dystrophy Association has announced it has awarded $1.6 million to Asklepios, a biotechnology company closely allied with the University of North Carolina at Chapel Hill, to develop gene therapy strategies for Duchenne muscular dystrophy (DMD), a fatal, childhood-onset disease.
Despite a roller-coaster ride of ups and downs during the past 15 years, gene therapy has continued to attract many of the world's brightest scientists. They are tantalized by the enormous potential that replacing missing genes or disabling defective ones offers for curing diseases of many kinds.
A consortium which includes Oxford researchers has won £1.6m in government funding for ground-breaking research into a possible cure for muscular dystrophy.