Source: Nature Medicine
Date: February 24, 2008
Summary:
A new way to manipulate human embryonic stem cells offers hope for an eventual cell-based therapy to treat muscular dystrophies. Muscular dystrophies, such as Duchenne’s muscular dystrophy (DMD), are caused by genetic mutations that lead to a loss of expression of dystrophin, a key structural protein of muscle cells, which results in cell dysfunction. When this occurs the cells can no longer regenerate after injury, resulting in progressive muscle weakness and eventual death. One hope for therapy has been to replenish these defective cells with ESCs that produce normal dystrophin).
