making muscle wasting history

According to a research by _Prof.Luis Garcia and Prof. Yvan Torrente

Stem cells, mainly satellite cells, were obtained from muscle bioptic material of Duchenne boys whose dystrophin gene had a deletion of exons 49 and 50. For their experiments, the Franco-Italian researchers used only those about 1% of the cells which contained the marker protein CD133 in their membranes: These cells had been shown earlier to be able to repair muscle cells and form new ones in damaged muscle tissue. The CD133+ cells
were multiplied in cell culture in the laboratory and then treated with a transforming vector of lentiviruses which carried in their genetic material genes of two antisense oligoribonucleotides for the skipping of exon 51 and it’s done in a similar way also for the genetic skipping of exon 23 in mdx mice.

The results showed a better muscle regeneration, a large amount of the expected dystrophin in the regenerated fibers
without the amino acids determined by exons 49, 50 and 51, an amelioration of muscle morphology (their structure), and a significantly restored muscle function. However, before clinical trials can be contemplated, the exact mechanism of exon skipping with lentiviral vectors will have to be understood completely, because these viruses with their charge enter the
genetic material of the muscle cells in a random way, and
possibly could disturb other genes or even induce tumors

The main goal is to combine gene modification strategies with cell-mediated therapies. This approach could permit autologous transplantation of cells, minimizing the risk of implant rejection. The results of this research shows that the combination of gene and stem cell approaches seems to be most promising, particularly intra-arterial injections of the patient’s own stem cells transduced by antisense oligonucleotide technology. This approach should offer the chance to distribute the autologous corrected stem cells to the whole body musculature providing a clinical benefit for dystrophic patients.