Duchenne’s Muscular Dystrophy, or DMD, has been in the news a lot recently. It’s the most common form of muscular dystrophy and was most recently featured in the Right to Try fight. The FDA also approved the first treatment of DMD’s symptoms in 2017, but a new paper published in Science points to a possible treatment of the root cause of the disorder. Scientists used a system-wide application of CRISPR to efficiently restore dystrophin expression in four dogs, a result that “exceeded [the lead author’s] most optimistic expectations.” There’s a ton of hurdles this treatment would have to pass to be a real therapy in humans, but this could be huge for DMD patients. Also, hey, it’s Labor Day weekend, maybe consider donating to the Muscular Dystrophy Association for Jerry’s Kids.
Two studies released this week looked at the tumor-suppressing gene p53 and found that it doesn’t play nicely with CRISPR-Cas9. P53 is responsible for scrambling emergency services when DNA is damaged, which CRISPR-Cas9 does when cutting into DNA strands and adding some new DNA. The emergency response is a take-no-prisoners approach which either ‘fixes’ the DNA, rendering the gene therapy useless, or kills the cell. Astute readers may notice this also makes the therapy useless. That could answer why gene editing can be inefficient, and that’s also where the cancer risk comes in. The only cells that survive this process have faulty p53 genes, thus compromising the cells’ ability to fight future tumors. This was only observed with the DNA insertion process, so don’t sound the death knell for CRISPR just yet.
While you may struggle to keep the extra pounds from coming on this holiday season, pigs don’t really have a choice. They apparently lack a gene called UCP1, which helps most mammals to regulate their body temperatures in cold weather. So the next best thing is typically insulation via packing the fat on, but this costs farmers a ton in feed, not to mention heating costs. Sensing an opportunity, researchers from the Chinese Academy of Sciences in Beijing used CRISPR/Cas-9 gene editing to give pigs a mouse version of the gene. The result? Healthy piglets with 24% less fat as compared to competing brands. That could mean happier pigs, that cost less, and are healthier to eat. No word yet on how CRISP-y the bacon was.
Ok, we may not exactly be at the point where we’re determining employment by genetic status, but CRISPR still gives some ethicists pause when it comes to human applications. However, a team of researchers led by Shoukhrat Mitalipov—whose greatest hits include creating “three-parent” monkeys and a technique for creating stem cells out of skin cells—decided they weren’t gonna let China have all the fun with pushing ethical boundaries. The team’s work culminated in the first gene-editing of a human embryo performed on US soil. They also did it better than their Chinese counterparts have been able to so far, with fewer unintended errors in portions of the DNA that weren’t being actively operated on.