New gene therapy for muscular dystrophy offers hope

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles. The research focuses on delivering a series of protein packets inside shuttle vectors to replace the defective DMD gene within the muscles.The gene for dystrophin is one of the largest in the human genome, and is difficult to fit inside a delivery shuttle. Instead of one shuttle, the new gene therapy uses a series of shuttles which take parts of the therapeutic gene inside the muscles, along with embedded instructions to begin assembling the fragments once within the body. The assembled genetic code will then start producing dystrophin, the protein lacking in patients with muscular dystrophy.