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New research from The Ottawa Hospital and the University of Ottawa has turned the understanding of Duchenne muscular dystrophy on its head.
The study, published Monday in Nature Medicine, showed for the first time that the deadly disease directly affects muscle stem cells.
The finding not only changes the medical and scientific world’s understanding of the disease but could eventually lead to more effective treatment, said Dr. Michael Rudnicki, senior author of the study. Rudnicki is director of the regenerative medicine program at The Ottawa Hospital and a professor at the University of Ottawa. He holds the Canada Research Chair in Molecular Genetics.
For Debra Chiabai, whose 15-year-old son Alex has the disease, the discovery offers hope.
“I am thrilled,” she said. “It is really important that we understand the disease. The better we understand the disease, the better we can target treatments.”
Duchenne muscular dystrophy is the most prevalent form of the disease, affecting one in 3,600 boys. Those affected experience progressive muscle weakness and usually do not live beyond their 20s or 30s.
Duchenne muscular dystrophy is caused by genetic mutations that result in the loss of dystrophin protein. Dystrophin protein was long believed to be a simple structural protein only found in muscle fibres. Rudnicki’s and his team’s research found that muscle stem cells also express the protein which results in their producing 10 times fewer precursor cells than do healthy stem cells.
Precursor cells become the fibres that make up muscles, which are then repaired after injuries and exercise. Without dystrophin, muscle stem cells cannot divide properly and repair damaged muscle.
The research was conducted on mouse cells. Rudnicki said he is optimistic work on human cells will come up with similar findings.
The research has uncovered “a whole new side of the disease we didn’t understand,” he added.
It’s implications are huge in terms of finding new treatments for the disease, now treated mostly with steroids.
“We believe it is theoretically possible to identify small drugs to modulate behaviours of the stem cells,” said Rudnicki. “This is really a paradigm-shifting discovery.”
Chiabai, whose son is participating in a drug trial in addition to being on steroids, said the discovery offers a number of possibilities for future treatments. New drugs could be developed, based on the updated understanding of the disease, something that would take years. She is also hopeful drugs that have already been approved for other diseases that might treat Duchenne muscular dystrophy.
“Anything that can give us hope is a good thing, and now we understand more than we did before.”
Rudnicki noted that the game-changing discovery validates the importance of basic, or exploratory research. Research funding in Canada has increasingly shifted toward applied research, leaving less money and support for “curiosity driven research” that can help “radically change the way we think about a disease.”
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The study, published Monday in Nature Medicine, showed for the first time that the deadly disease directly affects muscle stem cells.
The finding not only changes the medical and scientific world’s understanding of the disease but could eventually lead to more effective treatment, said Dr. Michael Rudnicki, senior author of the study. Rudnicki is director of the regenerative medicine program at The Ottawa Hospital and a professor at the University of Ottawa. He holds the Canada Research Chair in Molecular Genetics.
For Debra Chiabai, whose 15-year-old son Alex has the disease, the discovery offers hope.
“I am thrilled,” she said. “It is really important that we understand the disease. The better we understand the disease, the better we can target treatments.”
Duchenne muscular dystrophy is the most prevalent form of the disease, affecting one in 3,600 boys. Those affected experience progressive muscle weakness and usually do not live beyond their 20s or 30s.
Duchenne muscular dystrophy is caused by genetic mutations that result in the loss of dystrophin protein. Dystrophin protein was long believed to be a simple structural protein only found in muscle fibres. Rudnicki’s and his team’s research found that muscle stem cells also express the protein which results in their producing 10 times fewer precursor cells than do healthy stem cells.
Precursor cells become the fibres that make up muscles, which are then repaired after injuries and exercise. Without dystrophin, muscle stem cells cannot divide properly and repair damaged muscle.
The research was conducted on mouse cells. Rudnicki said he is optimistic work on human cells will come up with similar findings.
The research has uncovered “a whole new side of the disease we didn’t understand,” he added.
It’s implications are huge in terms of finding new treatments for the disease, now treated mostly with steroids.
“We believe it is theoretically possible to identify small drugs to modulate behaviours of the stem cells,” said Rudnicki. “This is really a paradigm-shifting discovery.”
Chiabai, whose son is participating in a drug trial in addition to being on steroids, said the discovery offers a number of possibilities for future treatments. New drugs could be developed, based on the updated understanding of the disease, something that would take years. She is also hopeful drugs that have already been approved for other diseases that might treat Duchenne muscular dystrophy.
“Anything that can give us hope is a good thing, and now we understand more than we did before.”
Rudnicki noted that the game-changing discovery validates the importance of basic, or exploratory research. Research funding in Canada has increasingly shifted toward applied research, leaving less money and support for “curiosity driven research” that can help “radically change the way we think about a disease.”
查看原文...
