In the last few weeks, excitement regarding a potential new approach (CRISPR/Cas9) to treating DMD has grown. Science recently published several individual labs' work demonstrating the ability to correct the basal genetic defect in DMD in the mouse model of DMD. These exciting results have been shared in several prominent media outlets, including the New York Times, The Guardian, and the BBC.
Though I'm no expert, CRISPR/Cas9 certainly is one of the most revolutionary developments in science and medicine in the last year, and will be something to keep a sharp eye on moving forward. Different than all previous therapies, CRISPR/Cas9 edits the actual genome (DNA), rather than transcriptome (RNA), or protein, leading to permanent changes within the host. This opens up many more questions than answers. Is it ethical to modify the genome or, how can this be used to treat patients with unique and individual mutations?
Unleashing the capacity of CRISPR/Cas9 is leading to a new epoch of science and medicine, and it will be exciting to see how we can utilize it to treat the muscular dystrophies.
I'm a current MD-PhD candidate, working hard to help treat and manage muscular dystrophies