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Autologous Transplantation of Muscle-Derived CD133+ Stem Cells in Duchenne Muscle Patients

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Abstract:

Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive muscle disease due to defect on the gene encoding dystrophin. The lack of a functional dystrophin in muscles results in the fragility of the muscle fiber membrane with progressive muscle weakness and premature death. There is no cure for DMD and current treatment options focus primarily on respiratory assistance, comfort care, and delaying the loss of ambulation. Recent works support the idea that stem cells can contribute to muscle repair as well as to replenishment of the satellite cell pool. Here we tested the safety of autologous transplantation of muscle-derived CD133+ cells in eight boys with Duchenne muscular dystrophy in a 7-month, double-blind phase I clinical trial. Stem cell safety was tested by measuring muscle strength and evaluating muscle structures with MRI and histological analysis. Timed cardiac and pulmonary function tests were secondary outcome measures. No local or systemic side effects were observed in all treated DMD patients. Treated patients had an increased ratio of capillary per muscle fibers with a switch from slow to fast myosin-positive myofibers.

Keywords: Autologous stem cell transplantation; CD133; Muscular dystrophy

Document Type: Research Article

DOI: http://dx.doi.org/10.3727/000000007783465064

Affiliations: 1: Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy 2: Department of Experimental Medicine, Human Physiology Unit, University of Pavia, Pavia, Italy 3: Centro Trasfusionale e di Immunologia dei Trapianti, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy 4: Stem Cell Research Institute, San Raffaele Hospital, Italy 5: Department of Surgery, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy 6: Department of Anesthesia and Critical Care Medicine, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy 7: Radiology Unit, Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, University of Milan, Italy 8: IRCCS Eugenio Medea, Bosisio Parini, Italy 9: ‡CNRS, Cytoskeleton and Development, Paris, France 10: *Fondazione IRCCS Ospedale Maggiore Policlinico of Milan, Department of Neurological Sciences, Dino Ferrari Center, University of Milan, Italy, ††IRCCS Eugenio Medea, Bosisio Parini, Italy

Publication date: June 1, 2007

More about this publication?
  • Cell Transplantation publishes original, peer-reviewed research and review articles on the subject of cell transplantation and its application to human diseases. To ensure high-quality contributions from all areas of transplantation, separate section editors and editorial boards have been established. Articles deal with a wide range of topics including physiological, medical, preclinical, tissue engineering, and device-oriented aspects of transplantation of nervous system, endocrine, growth factor-secreting, bone marrow, epithelial, endothelial, and genetically engineered cells, among others. Basic clinical studies and immunological research papers are also featured. To provide complete coverage of this revolutionary field, Cell Transplantation will report on relevant technological advances, and ethical and regulatory considerations of cell transplants. Cell Transplantation is now an Open Access journal starting with volume 18 in 2009, and therefore there will be an inexpensive publication charge, which is dependent on the number of pages, in addition to the charge for color figures. This will allow work to be disseminated to a wider audience and also entitle the corresponding author to a free PDF, as well as prepublication of an unedited version of the manuscript.
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