Scientists may have found a possible cure for a disorder that is common in ten year-old boys. Using a new gene-editing technique, called Crispr-Cas9, they experimented on lab rats. They have not yet tried it on humans, but they are hopeful that it will work.
The Crispr-Cas9 technique is a way of cutting out the defective part of a gene. The defective part is a stretch of DNA called an exon. When the exon is cut out, the muscle cells are able to make a protein that will increase muscle strength. This worked on mice. The next step is to see if it will work on humans.
Three teams, from Duke, Harvard, and the University of Texas Southwestern Medical Center, were working individually on this cure. They discovered it around the same time, on December 31, 2015. They were not the only groups researching this idea, but they were the first to prove that it works, at least on mice. Scientists are not sure how the human immune system will react to this technique. They do know that it will take a lot of time to get to that stage.
The people to which this breakthrough is targeted are patients with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy is caused by a gene defect, which affects a protein called dystrophin that helps your muscles function. It affects 1 out of every 3500 boys younger than ten, forcing them into wheelchairs before they’re even pre-teens, and putting them at risk of breathing problems or early death. Scientists have been working on finding a cure for a long time, with no luck. Now that this technique is proven to work, there is hope that it could work on other diseases too.
I chose this article because I didn’t know much about gene-editing, or about genes at all, and I wanted to learn more. I found it on a New York Times website under science. I learned a lot from it, including what genes are, how they affect who we are, and the process of gene-editing. I also learned about Duchenne Muscular Dystrophy, which I hadn’t heard of before. It connects with our unit because it is about health in humans, and the science behind gene-editing.
I think even the idea of gene-editing is a huge step in the right direction for science and cures. It is a game-changer for everybody who has or is researching Duchenne Muscular Dystrophy, other diseases that could possibly be treated by this technique. Though there is still a lot of work that has to be done, this is definitely good news.