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 a healthy copy of the gene dystrophin into mice with a condition that mimics muscular dystrophy. The dystrophin gene is mutated and as a result produces a defective protein in the roughly 20,000 people in the United States with the most common form of the disease. Using gene therapy to treat muscular dystrophy isn't a new idea. Thomas Rando, MD, PhD, associate professor of neurology and neurological sciences, said that researchers have tried several different techniques with variable success. One hurdle is getting genes into muscle cells all over the body. Another is convincing those cells to permanently produce the therapeutic protein made by those genes. The gene therapy technique Rando and postdoctoral fellow Carmen Bertoni, PhD, used was developed by Michele Calos, PhD, associate professor of genetics. One of the main advantages of this method is that it could potentially provide a long-term fix for a variety of genetic diseases, including muscular dystrophy.

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 8340 - Posted: 12.29.2005

Young blood could help revive tired ageing muscles, researchers suggest. Old people's muscles are known not to heal in the same way young people's do, but a Stanford University team suggests it is old blood that is to blame. The study found special stem cells come to the rescue of damaged young muscles, but are not triggered in older ones. Writing in Nature, the team say tests on mice suggest something in young blood spurs the stem cells into action to repair the muscle damage. It had been recognised that old muscles had the capacity to repair themselves, but that - for some reason - they failed to do so. The Stanford researchers focussed on muscle stem cells, called satellite cells, that are spread throughout muscle tissue. In young mice and humans, the cells come to life if they are needed to repair damaged muscle. But the team found that they fail to come to the rescue of older muscle - even though they are still present. In their tests, the team surgically connected the circulatory systems of an old mouse with that of a young one, or to another old mouse. They then damaged muscle in the older mice. If old mice were connected to young ones, and therefore had 'young blood' flowing through their bodies, healed normally. However, when old mice were connected to other old mice, and were sharing old blood, they healed slowly. (C)BBC

Related chapters from BN: Chapter 11: Motor Control and Plasticity; Chapter 5: Hormones and the Brain
Related chapters from MM:Chapter 5: The Sensorimotor System; Chapter 8: Hormones and Sex
Link ID: 7313 - Posted: 05.07.2005

Early treatment with a drug can delay the onset and progression of heart failure in children with Duchenne muscular dystrophy, research suggests. DMD is an incurable genetic disease causing muscle wastage, which often leads to fatal cardiac problems. The study shows the drug, perindopril, can slow heart muscle degeneration - and thus ward off heart failure. The research by Paris's Cochin Hospital involved 57 children, says the Journal of the American College of Cardiology. Lead researcher Professor Denis Duboc said: "For the first time, we have shown that it is possible to slow progression in this rare degenerative disease. "In DMD, the heart muscles are affected and cardiac problems are fatal in around 40% of children." The five-year study focused on the effect on perindopril, a drug from a class known as ACE inhibitors, widely used to treat high blood pressure and heart failure. Some 57 children with DMD received either perindopril, or a dummy drug. Eight in the dummy group went on to develop signs of heart failure, and three died from the condition. In contrast, just one of the perindopril group showed signs of heart failure, and none died from the condition during the study. Professor Duboc said the results suggested early treatment with perindopril might also benefit other people genetically predisposed to heart failure. DMD, one of the most common forms of muscular dystrophy, is caused by a lack of a protein called dystrophin which helps keep the muscles intact. It strikes children at a young age, and affects almost exclusively boys who rarely survive beyond their early 30s. (C)BBC

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 7052 - Posted: 03.20.2005

A study on mice suggests that a type of stem cells found in blood vessels may someday be able to regenerate wasting muscle in muscular dystrophy (MD) patients. The authors caution that more research must be done before researchers consider applying these findings to humans. Nonetheless, their results provide a possible new direction for efforts that have met largely with frustration thus far. The study appears in the journal Science, published by AAAS, the science society. The research team, led by Giulio Cossu of the Stem Cell Research Institute, in Milan, and the University of Rome and the Institute of Cell Biology and Tissue Engineering, in Rome, has found that these stem cells can cross from the bloodstream, into muscle tissue. There, they seem to take on a new identity, helping to generate new muscle fibers in mice with MD-like symptoms. MD is a collection of disorders caused by genetic defects that lead to increasing muscle weakness over time. These disorders currently have no cure.

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 4026 - Posted: 07.11.2003

Scientists are encouraged by the early success of treatment which may eventually help patients with a form of muscular dystrophy. Duchenne muscular dystrophy is a wasting disease caused by mutations on a particular gene. It is the most common muscular dystrophy, affecting one in 3,500 children - most of whom die early in life as a result. The mutations on the gene stop it producing the chemical needed to protect muscle cells and prevent wasting. Some experts believe that it may be possible to alleviate the disease by replacing the gene entirely. However, a slightly different strategy has paid dividends for researchers at the Medical Research Council's Clinical Sciences Centre Instead of trying to insert an entirely new version of the gene - called the dystrophin gene - which is problematic simply because of its large size, scientists are trying to issue the body instructions to ignore the faulty bits. While this, if successful, does not completely correct the problem, it does mean that a body chemical is produced that is almost as effective as the normal version. The technique, called "anti-sense" therapy, might also be easier to get working in a drug than full-blown gene therapy. (C) BBC

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 4002 - Posted: 07.08.2003

Jane Elliott, BBC News Online Health Staff When Catherine Crossin started suffering from fever, aching and exhaustion she thought she just had a bad case of flu. She took to her bed and waited for the illness to pass. Medics agreed it was probably flu, but soon her condition deteriorated, leaving her paralysed and unable to move even her little finger. She was so ill she needed 11 weeks in hospital to recover. "I couldn't bear anyone to touch me, I was so sensitive. My skin felt as if it was on fire." Her body became badly swollen and she was unable to move her muscles. For a while everyone was in the dark about Mrs Crossin's disease and she had a barrage of tests. "I had always been so healthy. I had never had colds and now I could not do anything," she said. Then doctors found she had a very serious case of polymyositis. Myositis is a relatively uncommon condition, which only affects about five new cases a year for every million people, leaving their muscles badly inflamed. (C) BBC

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 3531 - Posted: 03.08.2003

The discovery of genes that control the development of muscles in the fruit fly could help unravel the secrets of a devasting human disease. Cachexia is a severe wasting disorder normally linked to advanced cancer, Aids and a variety of chronic infections. It causes not just the loss of fat, but also of bone and muscle, and happens regardless of the amount the patient manages to eat. The patient's metabolism speeds up, burning more calories. The condition further robs patients of the ability to fight the disease which triggered the cachexia. It is believed that chemicals called cytokines released by the body in response to the underlying illness are responsible for the wasting illness. (C) BBC

Related chapters from BN: Chapter 13: Homeostasis: Active Regulation of the Internal Environment; Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 9: Homeostasis: Active Regulation of the Internal Environment; Chapter 5: The Sensorimotor System
Link ID: 3394 - Posted: 02.03.2003

NASA researchers are learning new things about the human brain by studying how astronauts regain their balance. Balancing is not as easy as it seems--just try to stand on one foot for a full minute, and you'll get a sense of the constant effort involved. It's one of those complex skills like reading that becomes so automatic with practice, we simply forget how tricky they were to learn. And, like reading, you might suppose it would take something extraordinary to make you forget. Indeed it does. Like traveling to space. Researchers have found that astronauts who return from a space voyage can still balance, but they find it far more difficult. That's because, explains NASA neuroscientist Bill Paloski, their brains are no longer sure how to interpret the information that comes from their senses.

Related chapters from BN: Chapter 9: Hearing, Balance, Taste, and Smell; Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 6: Hearing, Balance, Taste, and Smell; Chapter 5: The Sensorimotor System
Link ID: 3092 - Posted: 12.02.2002

by Dennis Meredith Steven Vogel was suffering sore muscles -- ironic for a biologist who had just published a widely praised book on the science and history of muscles, from flies to humans. Ensconced in his comfortable office, the sinewy, fit scientist-author of Prime Mover: A Natural History of Muscle (Norton, 2002) revealed that he had been persuaded to walk down the Eiffel Tower. Ever the scientist, Vogel precisely explained the basis of his discomfort. "You're exerting more force when you decelerate them when you accelerate, but the aerobic cost is so low you don't notice that you're doing much," said the James B. Duke Professor of Biology, wincing. "You don't notice it until afterwards." [Steven Vogel, a Duke biologist and author of Prime Mover: A Natural History of Muscle Photo: Les Todd/ Duke Photography] Indeed, the phenomenon of sore muscles is only one scientific morsel Vogel offers in a smorgasbord of topics covered in his book, including that facts that

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 2306 - Posted: 07.14.2002

A treatment which uses "master cells" from the muscles could eventually help patients with the muscle-wasting illness muscular dystrophy. The experiments - although so far only in mice - restored some muscle strength. People with muscular dystrophy do not have the ability to make a protein called dystrophin - which is vital to keep muscles working properly. Doctors used stem cells taken from the muscles of healthy newborn mice. These were then transplanted into the muscles of mice bred to simulate muscular dystrophy. Scientists found that the stem cells turned not only into muscle cells, but also into nerve and blood vessel cells. (C) BBC

Related chapters from BN: Chapter 11: Motor Control and Plasticity; Chapter 7: Life-Span Development of the Brain and Behavior
Related chapters from MM:Chapter 5: The Sensorimotor System; Chapter 4: Development of the Brain
Link ID: 2145 - Posted: 05.27.2002

By C. CLAIBORNE RAY Q. Why do chickens have white meat and dark meat and ducks have only dark meat? A. Chickens and ducks have different exercise habits and different muscle structures, with differing levels of a dark substance called myoglobin. Chickens, which are mostly earthbound, with an occasional spurt of flying, have breasts that contain mostly fast-twitch muscle fibers; these contract powerfully for a short hop in the air, but they soon become tired. Copyright 2002 The New York Times Company

Related chapters from BN: Chapter 11: Motor Control and Plasticity
Related chapters from MM:Chapter 5: The Sensorimotor System
Link ID: 1920 - Posted: 04.20.2002