Stem cells from human fat differentiate into muscle cells and produce dystrophin (green) when injected into mice.
DMD is caused by a lack of the cytoskeletal protein dystrophin, which provides muscle cells with structural stability. Several treatment strategies for DMD are under investigation, including gene therapy to replace the defective dystrophin gene, and stem cell transplantation to generate new dystrophin-producing muscle cells.
Previous work by this group and others revealed...
The Rockefeller University Press
2005
The Rockefeller University Press
2005
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