(Top) Immunofluorescence staining of the progenitor cells derived from
the pluripotent stem cells generated from one of the patients. Specifically, myotubes (the counterpart of muscle fibres in a dish)
from differentiated progenitor cells. Red: myosin heavy chain; blue: nuclei. (Bottom) Muscle progenitor cells (green) in vivo in a muscle three weeks
after transplantation in dystrophic mice.
Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published today in Science Translational Medicine . For the first time, scientists have turned muscular dystrophy patients' fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice. The new technique, which was initially developed at the San Raffaele Scientific Institute of Milan and completed at UCL, could be used in the future for treating patients with limb-girdle muscular dystrophy (a rare form in which the shoulders and hips are primarily affected) and, possibly, other forms of muscular dystrophies. Muscular dystrophies are genetic disorders primarily affecting skeletal muscle that result in greatly impaired mobility and, in severe cases, respiratory and cardiac dysfunction. There is no effective treatment, although several new approaches are entering clinical testing including cell therapy. This is a major proof of concept study.
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