Ideally, instating feasible dystrophin through genetic-based procedures would be the way to go, but gene therapies haven't been successful. Researchers tried replacing the gene that copies Becker's Muscle Dystrophy phenotype using adeno-associated virus vector. That was unsuccessful because the proteins produced a reaction from the immune system causing T cell-mediated cytotoxicity, against both the protein and the vector. Researchers also tried to suppress what they called a nonsense mutation. With this mutation the stop codon is placed too soon, stopping the dystrophin protein from being made. This method also failed. …show more content…
"(page2 paragraph1; lines 1-3)" The FDA determined that this was not a successful method after short-term and long-term trials were ran and neither yielded favorable results. The second trial consisted of twelve patients. Four which were on IV infusions of 30mg/kg, four on IV infusions if 50mg/kg and four who received placebo. The infusions included Eteplirsen, which after just one injection had increased dystrophin protein expressions. There was a significant increase in dystrophin positive fibers in this study. It showed a seventy-five-meter