Cell Transplantation and “Stem Cell Therapy” in the Treatment of Myopathies: Many Promises in Mice, Few Realities in Humans
Figure 2
Gene complementation after myoblast transplantation restored the expression of dystrophin in the myofibers of a DMD patient. The figure shows serial cross-sections of a muscle biopsy done in the gastrocnemius of a 26-year-old DMD patient, corresponding to a site that was transplanted 1 month before with myoblasts proliferated in culture from a muscle biopsy performed in the deltoid of his father. Sections were stained with hematoxylin and eosin, and for the fluorescent immunodetection of dystrophin they were with MANEX12-16 (an antibody that detects an epitope coded by the exons 12 to 16 of the dystrophin gene) and DYS2 (an antibody that detects an epitope coded by the exons 77 to 79 of the dystrophin gene). The section stained with hematoxylin and eosin shows a skeletal muscle with an intense substitution by adipose tissue, isolated groups of small-sized myofibers, and some fibrosis. The fluorescent immunodetection of dystrophin in the serial sections shows that an important proportion of these myofibers exhibited subsarcolemmal staining, both with an antibody that recognizes epitopes deleted in the patient (MANEX12-16, deletion was from exons 1 to 44) and with an antibody that recognizes the carboxy-terminal portion of the protein (DYS2). Both immunodetections were negative in the contralateral gastrocnemius injected with saline instead of normal myoblasts. Scale bars = 100 μm.
