dystrophy in Biological News

New therapy substitutes missing protein in those with muscular dystrophy

... disease and the most common form of muscular dystrophy in children. In the mouse model, researchers ... as a viable therapy for the treatment of muscular dystrophy as well as cardiac muscle diseases caused by loss ... Molecular Biology & Biophysics. Muscular dystrophy causes the muscles in the body to progressively ...

Muscular dystrophy diagnosis delayed almost 2.5 years in boys

... Boys show signs of Duchenne Muscular dystrophy (DMD) for 2 years before they obtain a diagnosis ... more children." DMD, the most common muscular dystrophy in children is a particularly devastating form of ... boys born since 1982 with DMD or Becker Muscular dystrophy in the ...

March of Dimes awards $250,000 prize to scientists unraveling the causes of muscular dystrophy

... ways to diagnose and potentially treat muscular dystrophy have been chosen to receive the 2009 March of ... are affected by the nine forms of muscular dystrophy and other related neuromuscular disorders. Among this group are facioscapulohumeral muscular dystrophy and limb girdle muscular dystrophy, as well as ...

American Chemical Society's Weekly PressPac -- Nov. 19, 2008

... of a long-sought drug to treat myotonic muscular dystrophy (MMD), the most common form of muscular dystrophy in adults. MMD affects about 1 in 8,000 people. ... Discovery of Lead Compounds Targeting Myotonic dystrophy (DM1)" DOWNLOAD FULL TEXT ARTICLE ...

Scientist clears hurdles for muscular dystrophy therapy

... form of muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common type of the disease, ... dystrophy." Patients with Duchnne muscular dystrophy have a gene mutation that disrupts the production ... to the hearts of newborn mice with muscular dystrophy and found that gene therapy corrected many of the ...

Study may explain exercise-induced fatigue in muscular dystrophies

... that occurs in people with many forms of muscular dystrophy is distinct from the inherent muscle weakness ... similar to the fatigue experience by muscular dystrophy patients after a short period of walking. "A ... said. Working with mouse models of muscular dystrophy and normal mice engineered to lack nNOS, the UI ...

Researchers discover molecular basis of a form of muscular dystrophy

... ) that people with limb-girdle muscular dystrophy are missing a protein called c-FLIP, which the ... to stop muscle wasting from limb-girdle muscular dystrophy and other conditions. Unfortunately, rare diseases like limb-girdle muscular dystrophy dont get the attention or funding they deserve, ...

UVA reports surprising findings related to myotonic muscular dystrophy

... shows that, in cases of Type 1 myotonic muscular dystrophy (DM1), a well known heart protein does several ... things. DM1 is the most common form of muscular dystrophy in adults and affects approximately 40,000 adults ... to potential therapies. Myotonic muscular dystrophy is recognized as the first example of a disease ...

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

... researchers have demonstrated that a muscular dystrophy patient should be able to maintain a normal ... are healthy. Patients with Duchenne muscular dystrophy and Becker muscular dystrophy have a gene mutation that disrupts the production ...

'Show me the money!' MDA greenlights grants

... and private funds for biomedical research have become scarce, the Muscular dystrophy Association reasserts its leadership in the fight against muscle diseases ... While advances in treatment of the devastating Duchenne muscular dystrophy (DMD) have prolonged boys' lives, physicians are seeing more heart disease ...

Stem cell breakthrough gives new hope to sufferers of muscle-wasting diseases

... are looking to launch human trials treating specific forms of muscular dystrophy such as oculopharyngeal dystrophy within the next three to five years. ...

Cell 'anchors' required to prevent muscular dystrophy

... to my pediatric textbook and saw images of people with a form of muscular dystrophy who had splayed shoulder bones," said Bennett, "This opened our eyes to ... to muscle cell organization. They knew that people with Duchenne muscular dystrophy were missing the protein dystrophin, and that dystrophin is needed for a ...

New clinical trial results show how personalized medicine will alter treatment of genetic disorders

... of a small clinical trial involving a new treatment for Duchenne muscular dystrophy provides a proof-of-principle for personalized molecular medicine. ... in small areas of muscle tissue at the injection site of muscular dystrophy patients. "Dozens of specific sequences will be required for ...

Sticky protein helps reinforce fragile muscle membranes

... muscular dystrophies include Fukuyama Congenital Muscular Dystrophy, Walker-Warburg Syndrome and Muscle-Eye-Brain disease and limb-girdle muscular dystrophy 2I. In these so-called dystroglycanopathies, too few sugar groups are added to alpha dystroglycan, leading to a version of the protein that does not ...

MIT: Jeremy Nathans to deliver Scolnick Prize lecture

... most common type of macular degeneration in children and young adults. Nathans and colleagues also determined that the vitelliform type of macular dystrophy is caused by abnormalities in a member of a previously unknown family of ion channels proteins that allow ions to enter and exit cells. ...

Ultrasound imaging now possible with a smartphone

... to the clinic, and the results would come back to the caregiver. A group at the Washington University Medical School studying Duchene's Muscular dystrophy is very interested in our devices and hope they can incorporate them into their research plans." Field trials in the Third World Richard and ...

Lamin A/C deficiency is 'unnerving'

... affected striated and skeletal muscle because Nesprin-1 and SUN2 are highly expressed in these tissues. Samples from Emery-Dreifuss muscular dystrophy patients exhibit similar hallmarks of skeletal muscle functional denervation, suggesting the authors are on the right track. ...

Potential therapy for congenital muscular dystrophy

... in a mouse model for alpha 7 integrin congenital myopathy," appears in the January 2009 issue of The American Journal of Pathology . Muscular dystrophy is a group of inherited genetic diseases that cause progressive muscle weakness. In one type of muscular dystrophy, patients with mutations in the ...

Single adult stem cell can self renew, repair tissue damage in live mammal

... then transplant skeletal adult muscle stems cells could have a wide impact in treating not only a variety of muscle wasting diseases such as muscular dystrophy but also severe muscle injuries or loss of function from aging and disuse. In other experiments, the researchers transplanted between 10 and 500 ...

The Burnham buzz

... Enzyme May Hold the Key to Muscular dystrophy A mutation in the gene coding the protein dystrophin has long been known to be associated with muscular dystrophy, but the role the protein plays ...

Sarcospan, a little protein for a big problem

... spotlight. Peter et al. now show that adding it to muscle cells might ameliorate the most severe form of muscular dystrophy. In Duchenne muscular dystrophy (DMD), the mutated dystrophin protein fails to anchor correctly to its membrane glycoprotein complex. And without this anchoring, muscle cells ...

Mediator in communication between neurons and muscle cells found

... control because of problems with neurons, muscle cells and/or their communication. Some reports have implicated a mutant MuSK as a cause of muscular dystrophy and autoantibodies (antibodies the body makes against itself) to MuSK have been found in the blood of some patients. Now that researchers know ...

PTC124 shows activity in cystic fibrosis; Phase 2 proof-of-concept data published in Lancet

... the potential applicability of PTC124 to multiple genetic disorders, we have a pivotal study of PTC124 for nonsense-mutation Duchenne/Becker muscular dystrophy ongoing and are planning proof-of-concept studies in additional genetic disorders." ...

Robotics research: Enhancing the lives of people with disabilities

... and any remaining physiological information in their limbs, such as an electromyographic signal produced in muscles, could ultimately assist muscular dystrophy patients regain significant use of there limbs. "Better orthotic technologies could ultimately help people suffering from this disease greatly ...

RNA emerges from DNA's shadow

... trials that could lead to it becoming generally available. Results so far indicate dramatic improvements in both cystic fibrosis and muscular dystrophy sufferers, although it is only suitable for those disorders caused by the presence of a premature stop sign in a gene sequence, as a result of a ...

A new method to identify mutated genes in human diseases

... the ones of mutated genes in similar diseases. The study identified candidate genes for 81 diseases, including various forms of epilepsy and muscular dystrophy As in all such analyses, the results must be verified experimentally. However, the task of understanding the molecular basis of many diseases ...

Overexcited neurons not good for cell health

... (Muscle cells in the case of this study.) Many different diseases and conditions, such as many neurodegenerative diseases, certain cancers, muscular dystrophy and the aging process, cause loss of muscle cell function. How that happens is not well understood. We may have discovered an unexpected basis for ...

Purdue researchers obtain a snapshot clarifying how materials enter cells

... ABC proteins in humans and have found that more than a dozen disease states are associated with malfunctions of these proteins, including macular dystrophy and problems in the regulation of cholesterol and insulin secretion. Chen's team isolated ABC proteins from an E. coli bacterium, which is the ...

Pittsburgh scientists identify human source of stem cells with potential to repair muscle

... you would have if you took the stem cells from another source." Working in dystrophic mice while searching for a cure for Duchenne muscular dystrophy (DMD), Dr. Huard's laboratory team first identified a unique population of muscle-derived stem cells with the ability to repair muscle 8 years ago. ...

Switching genes to overdrive improves muscular dystrophy symptoms in mice

... Iowa College of Medicine. Duchenne muscular dystrophy (DMD) is the most common type of muscular dystrophy in children, occurring once in about every 5,000 ... mice with female MDX mice (the muscular dystrophy gene is carried by females in mouse and in ....

Scientists show drug can counteract muscular dystrophy in mice

... deacetylase, in two mouse models of muscular dystrophy (MD): one that naturally develops a disease similar to Duchenne muscular dystrophy in humans, the other genetically altered to develop a form of dystrophy similar to the human limb-girdle muscular ...

Experimental cancer drugs counter muscle deterioration seen in muscular dystrophy

... weakness and fiber deterioration seen in muscular dystrophy can be countered by a class of drugs currently ... muscle function declines in Duchenne muscular dystrophy and related dystrophies. Indeed, muscles ... to normal, non-dystrophic mice. Muscular dystrophy is a group of more than 30 genetic diseases, ...

Toxic molecule may cause most common type of muscular dystrophy

... 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 ... function of the body's cells. Myotonic muscular dystrophy is the first example of a disease caused by toxic ...

UF scientists test improved gene therapy method for hereditary heart conditions

... reverse symptoms in mice with a form of muscular dystrophy that damages the heart. They also tested the ... with Pompe disease, a rare form of muscular dystrophy that is usually fatal in the first year of life. ... including atherosclerosis, stroke, muscular dystrophy and an enlargement of the heart muscle known as ...

Mouse study: New muscle-building agent beats all previous ones

... than a related potential treatment for muscular dystrophy now in clinical trials. The new agent is a ... muscles are wasting due to diseases like muscular dystrophy or side effects from cancer treatment or AIDS. ... and whether it helps a mouse model of muscular dystrophy retain enough muscle strength to prolong life. ...

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

... disease. Using gene therapy to treat muscular dystrophy isn't a new idea. Thomas Rando, MD, PhD, ... repair the entire muscle cell. Previous muscular dystrophy gene therapy studies did not look at whether the ... that the road to a gene therapy cure for muscular dystrophy is still a long one. However, Calos is confident ...

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

... therapy for the treatment of congenital muscular dystrophy (CMD) in mice, demonstrating that the formidable ... ever being feasible for children with muscular dystrophy can be overcome. Moreover, their results, ... have shown that gene therapy for muscular dystrophy is both feasible and effective in a mouse model ...

Leading reason for corneal transplants comes into focus

... affects more than 4 percent of the population over 40. Late in life, the dystrophy causes swelling of the cornea and can severely affect vision, making it ... in FCD more than six years ago when he treated a woman with a corneal dystrophy of unknown origin that looked remarkably similar to FCD despite slightly ...

A giant among minnows: Giant danio can keep growing

... Other studies have already shown that a condition similar to muscular dystrophy can be experimentally induced in zebrafish. One of the next steps for the ... “We anticipate that the giant danio will be able to handle the muscular dystrophy better because of their ability to increase muscle,?Biga said. What makes ...