The Promising Future of Gene Therapy with Duchenne Muscular Dystrophy

Resizing large dystrophin genes is essential to the study since genetically engineered viruses can only carry so much load. Researchers worked to create mini-versions of rAAV and tested it to boys with Duchenne MD. The study reached a conclusion that it was safe but caused some unwanted immune responses to the test subjects. The mini-dystrophin protein, therefore, cannot be properly used by the muscle cells, limiting the effectiveness of this new approach. Another attempt at developing even smaller but fully functioning genes was made, thereby creating micro-dystrophin protein. As of now, this is an ongoing medical therapy currently undergoing the first phase of clinical testing.