Rule one of journalism is that if you have any plausible reason to mention Jennifer Lopez in a highly technical story about genetics and biochemistry, go for it! And so we have.
Lopez, according to the Hollywood Reporter, is hoping to produce a dramatic TV series titled “C.R.I.S.P.R.," which would feature male and female protagonists trying to save humanity from a mad scientist.
According to THR:
In the same vein of Castle, romance will blossom between the scientist and the FBI agent as they team to bring down a diabolical genius with a twisted God complex: her former boss. The drama will see mentor and protege battle for control over the human genome in a game of cat and mouse in which the future of our species may rest and all disease could one day be eradicated.
The bad scientist with the God complex apparently is devilish in his use of the gene-editing technique known as CRISPR (which stands for, as you may know by now, “clustered regularly interspaced short palindromic repeats"). NBC has asked for scripts, according to THR, but the network has yet to order a pilot, and so this show may yet evaporate, along with a million other dreams and aspirations, in the Southern California sunshine. (A spokesman for NBCUniversal told us by email, " 'CRISPR' is actually just in development, as you noted, and we don’t comment on those projects until they actually move along, if they do at all.")
Now, the news: Researchers at Yale and Carnegie Mellon universities have published a paper in Nature Communications revealing a completely different method of editing genes and targeting mutations that cause diseases. The new technique is not nearly as simple as CRISPR, but it's less likely to miss its target. That could make it potentially safer than CRISPR for gene therapy in human patients.
Although genetic manipulation has been around for decades, CRISPR has exploded in the last few years as a favored technique because it is versatile, inexpensive and relatively easy. Some of the key ingredients for this kind of gene editing can be ordered online and delivered by express mail. The CRISPR system was actually invented by bacteria in the distant past. Cells have their own complex systems for repairing the genome. CRISPR exploits a natural enzyme called Cas-9 that can target a section of a genetic code and snip out mutated or damaged segments.
The problem is that this is not yet a perfect science. CRISPR's genome-snipping isn't always in the right place. Scientists claim progress in limiting off-target effects, but CRISPR to this point hasn't matured enough as a technique to be used in human therapies.
“It's so good at cutting the genome, it tends to make cuts at the wrong place, too. I think our technology is much harder to make, but we believe it's much more specific, with less off-target effects,” said Peter Glazer, a Yale University geneticist and the co-author of the new study.
Glazer teamed with other scientists, including Danith Ly, a professor of chemistry at Carnegie Mellon, to develop a technique that takes synthetic genetic material — PNAs, for peptide nucleic acids — and injects it directly into the bloodstream of a mouse with a blood disease. The new study reports on the results of experiments on mice with the blood disease thalassemia, which is relatively common in humans and inhibits their levels of hemoglobin. Thalassemia is caused by a single mutation in the genetic code. The synthetic PNAs, injected into the mouse, are designed to bind with the mice DNA to form a kind of bump on the genome.
Indeed, instead of the usual double helix, that stretch of modified DNA will now have a triple helix. A cell has molecules that continually scan the DNA for something that doesn't look right, and “triplex DNA” is just such an anomaly. So the cell deploys its repair machinery to snip away the offending DNA segment. The laboratory technique developed by Glazer, Ly and their colleagues requires a second maneuver, the deployment of a DNA patch that contains a normal, non-mutant version of the hemoglobin gene. Ideally the DNA strand will repair itself by plugging in this non-mutant genetic patch.
This is tricky stuff and only works on a small percentage of cells. But — amazingly — even that level of efficiency is enough to suppress the diseases studied so far, the scientists say.
Major caveat: This has only been done in mice. Will it work in humans?
“That’s the goal, to try to replicate this mouse model in humans,” Ly told The Washington Post.
Now, back to Hollywood and its insatiable desire for stories about mad scientists. It was two centuries ago that Mary Shelley conjured up her tale of “Frankenstein,” and we are unlikely to escape the trope of the mad scientist anytime soon. But Glazer, for one, points out a fundamental truth about the human genome: It's really complicated. There's usually not a single genetic marker for physical traits or diseases.
“It's only the very obvious, single-gene disorders like sickle cell anemia where you have the obvious thing to edit,” Glazer said.
If someone asked him to create a human being who, for example, could run faster, he'd have no idea how to do it, he said.
The only people who know how to do something like that are the people in Hollywood.