Monday, November 27, 2006
Treatments For Muscular Dystrophy
Milan (Italy), 26 November: A potential stem cell treatment for muscular dystrophy is reported in a paper published online by Nature this week. The team transplanted a specific type of stem cell into the bloodstream of an animal model with the disease, demonstrating that they correct most of the dystrophic abnormalities.
Muscular dystrophy causes muscle degeneration, progressive paralysis and eventually death. Giulio Cossu and colleagues used mesoangioblasts in their study- stem cells, gathered from small blood vessels, that are 'programmed' to develop into muscle cells. They isolated these cells from normal and dystrophic dogs- that is, with an altered dystrophin protein. This animal is the only model system to present with the full spectrum of the disease pathology in humans. Stem cells isolated from dystrophic animals were modified to contain a corrected version of the dystrophin protein. The animals were injected five times at monthly intervals and improvements were seen in all cases. Those receiving unmodified donor cells showed a generally greater improvement than those injected with the corrected versions, but the potential success of using genetically corrected stem cells means that this form of transplant may be used on patients in the absence of life-long immune suppression treatment.
A News and Views article by Jeffrey Chamberlain reports that given the results in the paper, 'a fairly straightforward infusion of stem cells into the blood-stream might one day be able to treat muscular dystrophy.'
(ResearchSEA)
Muscular dystrophy causes muscle degeneration, progressive paralysis and eventually death. Giulio Cossu and colleagues used mesoangioblasts in their study- stem cells, gathered from small blood vessels, that are 'programmed' to develop into muscle cells. They isolated these cells from normal and dystrophic dogs- that is, with an altered dystrophin protein. This animal is the only model system to present with the full spectrum of the disease pathology in humans. Stem cells isolated from dystrophic animals were modified to contain a corrected version of the dystrophin protein. The animals were injected five times at monthly intervals and improvements were seen in all cases. Those receiving unmodified donor cells showed a generally greater improvement than those injected with the corrected versions, but the potential success of using genetically corrected stem cells means that this form of transplant may be used on patients in the absence of life-long immune suppression treatment.
A News and Views article by Jeffrey Chamberlain reports that given the results in the paper, 'a fairly straightforward infusion of stem cells into the blood-stream might one day be able to treat muscular dystrophy.'
(ResearchSEA)
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