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Dr. Giulio Cossu, director of the Stem Cell Research Institute at the San Raffaele Scientific Institute of Milan in Italy, announced this month promising results of stem cell transplantation into dogs affected by Duchenne muscular dystrophy. Two types of adult stem cells were compared, donor stem cells from healthy dogs and stem cells that were derived from the dystrophic dogs and then corrected through gene therapy.
These adult stem cells, called mesoangioblasts, can be harvested relatively easy from small blood vessels and can be grown in tissue culture and still maintain the ability to make muscle cells. The donor or corrected cells are then introduced into the dystrophic
dog via arteries and in the case of one dog, injected directly into the aorta of the heart.
The dog receiving the mesoangioblasts in the aorta was dramatically improved, walking well 5 months after the final treatment. The next most improved group was those dogs receiving the donor cells while undergoing immune suppression therapy. These dogs had almost normal levels of the dystrophin protein and their muscle function improved.
Although the stem cells that were taken from a dystrophic dog, corrected with gene therapy, and then re-introduced into that same dog, did not have as dramatic of an improvement, this technique warrants further study since a life long therapy of immune suppressants carries problems of their own. The gene therapy used human protein that was not full size, a “micro-dystrophin,” so additional research is warranted to see if changing these factors will improve the outcome.
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