Dystrophin News and Research

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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.

Discovery of stem cells that repair injured muscles

A University of Colorado at Boulder research team has identified a type of skeletal muscle stem cell that contributes to the repair of damaged muscles in mice, which could have important implications in the treatment of injured, diseased or aging muscle tissue in humans, including the ravages of muscular dystrophy.

5 Mar 2009

New findings raise questions about process used to identify experimental drug

A study by National Institutes of Health (NIH) researchers has revealed surprising new insights into the process used to initially identify an experimental drug now being tested in people with cystic fibrosis and muscular dystrophy.

3 Feb 2009

Proteins called ankyrins required to prevent muscular dystrophy

A protein that was first identified for playing a key role in regulating normal heart rhythms also appears to be significant in helping muscle cells survive the forces of muscle contraction.

13 Jan 2009

Adding protein sarcospan to muscle cells might help Duchenne muscular dystrophy

The overlooked and undervalued protein, sarcospan, just got its moment in the spotlight.

4 Nov 2008

New type of gene manipulation therapy offers hope for Duchenne muscular dystrophy sufferers

A medical research team at Carolinas Medical Center in Charlotte headed by Qi Long Lu, M.D., Ph.D., has made a discovery that holds promise to restore muscle function in patients afflicted by Duchenne muscular dystrophy (DMD).

21 Sep 2008

Proof-of-concept that purified muscle stem cells can be used in therapy

By injecting purified stem cells isolated from adult skeletal muscle, researchers have shown they can restore healthy muscle and improve muscle function in mice with a form of muscular dystrophy. Those muscle-building stem cells were derived from a larger pool of so-called satellite cells that normally associate with mature muscle fibers and play a role in muscle growth and repair.

10 Jul 2008

Investigational drug Debio-025 tested for preventing muscle fiber death in muscular dystrophy

An investigational antiviral drug currently undergoing human trials in Europe for treating Hepatitis C infections may have potential to reduce muscle cell damage in Duchenne and other forms of muscular dystrophy (MD).

17 Mar 2008

Long-term muscle improvements shown in gene therapy study in mice

Injecting a gene responsible for making a specific protein into a mouse that's used as a model for muscular dystrophy can lead to long-term improvements in the animal's muscle size and strength, a new study shows.

14 Mar 2008

Long-term muscle improvements shown in gene therapy study in mice

11 Mar 2008

Stem cell therapy restores muscle function in mice with muscular dystrophy

Scientists in Texas have been able to restore muscle function in mice with muscular dystrophy by using embryonic stem cells.

21 Jan 2008

Stem-cell transplantation improves muscles in MD animal model

Using embryonic stem cells from mice, UT Southwestern Medical Center researchers have prompted the growth of healthy - and more importantly, functioning - muscle cells in mice afflicted with a human model of Duchenne muscular dystrophy.

21 Jan 2008

Personalized medicine will alter treatment of genetic disorders

One of the nation's pre-eminent genetic researchers, Eric Hoffman, PhD, of Children's Research Institute at Children's National Medical Center, predicts that in relatively short order, medicine's next innovation--individualized molecular therapies--will have the unprecedented ability to treat muscular dystrophies, and other disorders.

28 Dec 2007

Reprogrammed human adult stem cells rescue diseased muscle in mice

Scientists report that adult stem cells isolated from humans with muscular dystrophy can be genetically corrected and used to induce functional improvement when transplanted into a mouse model of the disease.

13 Dec 2007

Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart

Heart disease is the leading cause of death in the United States and greatly affects the quality and length of life for individuals with specific forms of muscular dystrophy.

1 Nov 2007

Oral medication restores missing protein in boys with muscular dystrophy

PTC124, an oral medication that changes the way muscle cells interpret genetic information, holds promise as a treatment for some patients with Duchenne muscular dystrophy (DMD), the Muscular Dystrophy Association ( http://www.mda.org/ ) announced today.

19 Oct 2007

Massive microRNA scan uncovers leads to treating muscle degeneration

Researchers have discovered the first microRNAs - tiny bits of code that regulate gene activity - linked to each of 10 major degenerative muscular disorders, opening doors to new treatments and a better biological understanding of these debilitating, poorly understood, often untreatable diseases.

18 Oct 2007

Myoendothelial cells identified as new human source of stem cells with potential to repair muscle

For the first time, scientists at Children's Hospital of Pittsburgh of UPMC have discovered a unique population of adult stem cells derived from human muscle that could be used to treat muscle injuries and diseases such as heart attack and muscular dystrophy.

5 Sep 2007

Innovative gene therapy research for muscular dystrophy

A major grant of approximately $10 million from the National Institutes of Health will help investigators at Columbus Children's Research Institute (CCRI) at Columbus Children's Hospital learn more about strategies to treat muscular dystrophy, potentially leading to two new gene therapy approaches.

3 Aug 2007

New target for muscular dystrophy drug therapy

Researchers at the University of Pennsylvania School of Medicine report how the gene for utrophin, which codes for a protein very similar to dystrophin, the defective protein in Duchenne muscular dystrophy (DMD), puts the brakes on its own expression in muscle cells, thereby suggesting a new target for treatment.

13 Jul 2007

Protein dysferlin critical for resealing heart muscle cell membranes

During vigorous exercise, heart muscle cells take a beating. In fact, some of those cells rupture, and if not for a repair process capable of resealing cell membranes, those cells would die and cause heart damage (cardiomyopathy).

4 Jul 2007