Muscular Dystrophy Treated with Umbilical Cord Blood Stem Cells

Journal of Translational Medicine, January 14, 2009

Scientists in Brazil have used adult stem cells harvested from umbilical cord blood to treat muscular dystrophy.

Rather than referring to one disease, the term "muscular dystrophy" actually refers to a group of hereditary disorders of genetic origin and varying severity, depending upon the degree to which the dystrophin gene is defective or absent. Located at Xp21, the dystrophin gene codifies dystrophin, an essential component in the protein complex that is responsible for the membrane stability of muscle cells. A complete absence of the gene causes more severe forms of the disease such as the Duchenne form (DMD), whereas the presence of a defective gene causes milder forms of the disease such as the Becker form (BMD).

In this study, which was condcuted by Dr. Tatiana Jazedje and colleagues at the Human Genome Research Center in Sao Paulo, Brazil, the scientists took CD34+ adult stem cells derived from umbilical cord blood and established co-cultures which combined the stem cells with myoblasts from a patient who had been diagnosed with DMD. The CD34+ stem cells were already known to differentiate into muscle cells and to express dystrophin in vivo, but Dr. Jazedje and her colleagues were the first to show that this particular progenitor cell is also capable of regenerating muscle dystrophin in vitro, as the stem cells were found to have differentiated into mature myotubes after 15 days, while dystrophin-positive regions were also detected through immunofluorescence analysis.

As the authors concluded in their article, "Our findings showed that umbilical cord blood CD34+ stem cells have the potential to interact with dystrophic muscle cells restoring the dystrophin expression of DMD cells in vitro. Although utilized within the context of DMD, the results presented here may be valid to other muscle-related therapy applications."