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New therapy substitutes missing protein in those with muscular dystrophy
May 27, 2009Researchers were able to repair muscle tissue in mouse model
Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.
In the mouse model, researchers were able to substitute for the missing protein - dystrophin, which forms a key part of the framework that holds muscle tissue together - that results in the disease, effectively repairing weakened muscle tissue.
Researchers injected dystrophic mice with a protein called utrophin - a very close relative of dystrophin - that was modified with a cell-penetrating tag, called TAT.
The study is the first to establish the efficacy and feasibility of the TAT-utrophin-based protein as a viable therapy for the treatment of muscular dystrophy as well as cardiac muscle diseases caused by loss of dystrophin.
The research is published in the May 26, 2009 issue of PLoS Medicine.
"This unique approach can replace the missing protein without the complexities of gene replacement or stem cell approaches," said James Ervasti, Ph.D., principal investigator of the study and a professor in the Department of Biochemistry, Molecular Biology & Biophysics.
Muscular dystrophy causes the muscles in the body to progressively weaken. Duchenne is the most common and severe form of childhood muscular dystrophy. About one of 3,500 boys are born with the crippling disease. Symptoms usually begin in children who are 2 to 3 years-old, most are in a wheelchair by age 12, and many who have the disease pass away by their late teens to early 20s. Current treatment, limited to corticosteroids, are minimally effective and can cause serious side effects.
Research underway to battle muscular dystrophy with gene therapy and stem cell treatment shows promise, but major hurdles must be overcome before these approaches are viable in human patients, Ervasti said.
Delivering treatment to every muscle cell via gene therapy or stem cells is difficult because muscle tissue makes up such a large portion of the human body. Furthermore, the immune system may reject the cell or gene treatment because patients would treat the newly introduced cells or genes as a foreign substance.
Ervasti's method may conquer both of those problems. Upon injection, the TAT-utrophin combination spreads around the entire body efficiently and is able to penetrate the muscle cell wall to substitute for missing dystrophin. Because every cell in the body makes utrophin naturally, TAT-utrophin circumvents immunity issues associated with other therapeutic approaches.
"Our protein replacement approach most directly and simply addresses the cause of Duchenne muscular dystrophy," Ervasti said.
This new method is not a cure for muscular dystrophy. Rather, it would be a therapy most likely administered on a regular basis. If the treatment works in larger animal models and humans, it's most likely researchers would develop a drug for patients. Ervasti is hopeful the therapy can move into human clinical trials within 3 years.
University of Minnesota
Researchers injected dystrophic mice with a protein called utrophin - a very close relative of dystrophin - that was modified with a cell-penetrating tag, called TAT.
The study is the first to establish the efficacy and feasibility of the TAT-utrophin-based protein as a viable therapy for the treatment of muscular dystrophy as well as cardiac muscle diseases caused by loss of dystrophin.
The research is published in the May 26, 2009 issue of PLoS Medicine.
"This unique approach can replace the missing protein without the complexities of gene replacement or stem cell approaches," said James Ervasti, Ph.D., principal investigator of the study and a professor in the Department of Biochemistry, Molecular Biology & Biophysics.
Muscular dystrophy causes the muscles in the body to progressively weaken. Duchenne is the most common and severe form of childhood muscular dystrophy. About one of 3,500 boys are born with the crippling disease. Symptoms usually begin in children who are 2 to 3 years-old, most are in a wheelchair by age 12, and many who have the disease pass away by their late teens to early 20s. Current treatment, limited to corticosteroids, are minimally effective and can cause serious side effects.
Research underway to battle muscular dystrophy with gene therapy and stem cell treatment shows promise, but major hurdles must be overcome before these approaches are viable in human patients, Ervasti said.
Delivering treatment to every muscle cell via gene therapy or stem cells is difficult because muscle tissue makes up such a large portion of the human body. Furthermore, the immune system may reject the cell or gene treatment because patients would treat the newly introduced cells or genes as a foreign substance.
Ervasti's method may conquer both of those problems. Upon injection, the TAT-utrophin combination spreads around the entire body efficiently and is able to penetrate the muscle cell wall to substitute for missing dystrophin. Because every cell in the body makes utrophin naturally, TAT-utrophin circumvents immunity issues associated with other therapeutic approaches.
"Our protein replacement approach most directly and simply addresses the cause of Duchenne muscular dystrophy," Ervasti said.
This new method is not a cure for muscular dystrophy. Rather, it would be a therapy most likely administered on a regular basis. If the treatment works in larger animal models and humans, it's most likely researchers would develop a drug for patients. Ervasti is hopeful the therapy can move into human clinical trials within 3 years.
University of Minnesota
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Possible help in fight against muscle-wasting disease
A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease, according to researchers at the University of Oregon and the University of Rochester School of Medicine and Dentistry in New York.
Exon-skipping drug prevents muscle wasting, maintains muscle function in dystrophin deficient mice
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.
To regenerate muscle, cellular garbage men must become builders
For scientists at the European Molecular Biology Laboratory (EMBL) in Monterotondo, Italy, what seemed like a disappointing result turned out to be an important discovery.
Zoo volunteers help explain mysteries of the genome
As the University of Leicester approaches the 25th anniversary of the discovery of DNA fingerprinting (September 10), Leicester geneticists interested in a particular type of DNA are receiving some help from an unusual band of assistants.
Small molecule inhibits pathology associated with myotonic dystrophy type 1
Researchers at the University of Illinois have designed a small molecule that blocks an aberrant pathway associated with myotonic dystrophy type 1, the most common form of muscular dystrophy.
Researchers identify new function for protein missing in Duchenne muscular dystrophy
Researchers at the University of Minnesota and National Institutes of Health have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy.
Sticky protein helps reinforce fragile muscle membranes
A new study by scientists at the University of Iowa shows why muscle membranes don't rupture when healthy people exercise.
Stem cell surprise for tissue regeneration
Scientists working at the Carnegie Institution's Department of Embryology, with colleagues, have overturned previous research that identified critical genes for making muscle stem cells.
Researchers make progress toward early identification of muscular dystrophy
The saying "Knowing is half the battle" is never more true than when discussing early treatment of disease. Muscular dystrophy is one such disease where patients can benefit from early treatment. Now, new research is moving doctors and scientists closer to disease diagnosis in advance of patient symptoms.
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