Scientists reported on Wednesday that adult stem cells isolated
from humans with muscular dystrophy can be genetically corrected
and used to induce functional improvement when transplanted into a
mouse model of the disease.
The research by a team of researchers from Italy and France,
published in the December issue of Cell Stem Cell,
represents a significant advance toward the future development of a
gene therapy that uses a patient's own cells to treat this
devastating muscle-wasting disease.
Scientists reported on
Wednesday that adult stem cells isolated from humans with muscular
dystrophy can be genetically corrected and used to induce
functional improvement when transplanted into a mouse model of the
disease.(Yahoo File Photo)
Duchenne muscular dystrophy (DMD) is a hereditary disease caused
by a mutation in the gene that codes for a muscle protein called
dystrophin. DMD is characterized by a chronic degeneration of
skeletal muscle cells that leads to progressive muscle weakness.
Although intense research has focused on finding a way to replace
the defective dystrophin protein, at this time there is no cure for
DMD.
The research team led by Yvan Torrente from the University of
Milan used a combination of cell- and gene-based therapy to isolate
adult human stem cells from DMD patients. Then they used agenetic
technique to repair the mutated dystrophin gene so that dystrophin
synthesis was restored.
Importantly, intramuscular or intra-arterial delivery of the
genetically corrected muscle stem cells resulted in significant
recovery of muscle morphology, function, and dystrophin expression
in a mouse model of muscular dystrophy.
"These data demonstrate that genetically engineered stem cells
represent a possible tool for future stem cell-based autograft
applications in humans with DMD," says Torrente. However, the
authors caution that significant additional work needs to be done
prior to using this technology in humans.?
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(Xinhua News Agency December 14, 2007)
