Abstract:

BACKGROUND: Duchenne muscular dystrophy (DMD), a well-known genetic lethal in humans, cannot be effectively cured at present. Recently, scholars have performed basic research and animal trials using stem cells for treatment of DMD, and also conduct significant clinical studies.
OBJECTIVE: To investigate the feasibility and safety of therapy in patients with DMD who received stem cell transplantation in sequence by observing the change of their motor function, muscle cytothesis and regeneration, expression of dystrophin and altering of depletion gene.
METHODS: An 8-years old boy with DMD was treated by stem cell transplantation in sequence in May 2009. Exon 13 was depleted by multiplex ligation-dependent probe amplification. Stem cell transplantation in sequence was as follows: First, intravenous umbilical cord mesenchymal stem cell (UC-MSC) transplantation; Second, intramuscular UC-MSC transplantation; finally, monoploid allogene hemopoietic stem cell transplantation. Sero-enzyme, implantation proof of donor HLA antigen, expression of depletion gene and dystrophin of myocyte membrane, motor function were detected after transplantation.
RESULTS AND CONCLUSION: By stem cell transplantation in sequence for DMD, depletion gene could be substituted. Dystrophin of myocyte membrane expresses; sero-enzyme steps down significantly; motor function is improved. It could prevent the progression of disease and patient’s condition is to be ameliorated continuously.