Gene editing probably won’t make the next Spiderman, but it’s possible that the world’s first gene-edited babies have been born. He Jiankui, a Chinese scientist, shocked the world and its ethical sensibilities by revealing last week that he used CRISPR to lower two twin girls’ risk of HIV infection. And a third baby is on the way. Less HIV is certainly something we can all get behind, especially when World AIDS Day was last week. But CRISPR is still brand-spanking new, and scientists are questioning the medical appropriateness of this use of gene editing when the consequences aren’t super well understood and possibly catastrophic. Even a CRISPR co-founder is calling for a moratorium on live-embryo testing. Is this a breakthrough like the first IVF birth, or a slippery slope? From Nature: six questions that remain.
While the name “Gene” might be trending in the wrong direction (it’s the 2,112nd most popular male name in 2018, down 243 spots from 2017), according to Google, the term “gene therapy” is not. However, “according to a review of recent research studies that measured US public opinion related to gene medicine, the public is not yet aware of gene medicine; is unfamiliar with the terms being used to describe the topic; and, when faced with multiple therapeutic options, is worried about making informed decisions.” Did you know there’s an association focused on gene therapies? (There is always an association.) Introducing the Alliance for Regenerative Medicines or ARM. If you’re at all interested in the topic they seem to have decent publications. If you’re not interested, then here is a very funny video on the topic.
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.