The possibilities are astounding for curing diseases before they happen. Of course, the ways it can be used for harm are equally astounding…
By David Sussin
It's now possible to edit your DNA.
You can't stretch out a single string of DNA nucleotides and grab a scalpel and make a cut. The edge of the blade is too big -- thousands of times too big.
If you were a strand of DNA, the scalpel blade coming down to make a cut would be the size of the Himalayas. Not a mountain in the Himalayas, but the entire 1,500 mile long mountain range.
DNA is microscopic. Actually, it's smaller than that. It's nanoscopic, literally the size of a molecule because, well, a single strand of DNA is a molecule. The edge of a knife blade is room enough for millions of strands, enough to encode entire living organisms.
Yet even at that unfathomably small size, science figured out a way to edit a single strand…
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The technology is called CRISPR. The term is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. The process uses RNA to guide a protein to a targeted spot on a strand of DNA.
The protein breaks the DNA, and when the strand naturally fixes itself, science can intervene to manipulate the results. This process can reprogram genes, replacing bad ones, introducing new ones, or just deactivating a gene -- turning it off.
The possibilities are astounding for curing diseases before they happen. Of course, the ways it can be used for harm are equally astounding.
Before it was even proven to work on humans, CRISPR caught the attention of the military. Specifically, the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense.
DARPA's stated mission is to prevent technological surprise threats to the U.S. and, in turn, create the same as weapons to attack the enemy. There's no more frightening technological surprise than the existence of gene-editing as a potential weapon.
In 2017, DARPA launched the Safe Genes program to get their arms around gene-editing technologies. They wanted to control the effects, be able to reverse unwanted genetic elements introduced into humans by enemies of the State. They hoped to develop anti-CRISPR molecules that disable CRISPR systems -- fail-safe mechanisms in a genetic-level war.
That's the stated goal.
But remember, DARPA always looks at the other side, too. If they are trying to disable enemy CRISPR attacks, they are at the same time researching how to launch their own.
The potential weaponization of CRISPR technology is terrifying. The obvious possibilities include modifying pathogens to make them more contagious and resistant to all known cures; or creating targeted biological agents that single out groups with specific genetic predispositions -- even down to a single person meant to be eliminated.
But what if DARPA uses the technology on its own citizens? Gene editing done on entire populations could be designed to control human intelligence, behavior, or physical characteristics. Human autonomy could be erased.
Imagine gene editing done as soon as babies are born. They'd never know life as it was "meant" to be. Actually, you don't have to imagine it. It's happening now.
In this case, it's for good.
At Children's Hospital of Philadelphia earlier this year, a baby boy named KJ was born with a rare, deadly disease. It's called CPS1. It's caused by mutations in the CPS1 gene, which tells the body how to produce the CPS1 enzyme.
It shows you just how fragile we are, because this one enzyme is critical to removing toxic ammonia from the body. Without it, ammonia builds to toxic levels in the blood. The condition is life threatening. Even if children survive, they can suffer severe neurological damage. But the statistics are dire -- more than half of the children born with a CPS1 deficiency die.
But CRISPR may change that.
Physicians at the hospital have been developing a form of CRISPR that can target an individual's liver and correct the CPS1 gene. And KJ was a lucky recipient. He's the first human being ever to receive a bespoke CRISPR gene-editing treatment.
Before this, CRISPR has been used to target categories of illness meant to help all patients with a given disease (in 2023, the FDA approved a CRISPR-based therapy for sickle cell). But the CPS1 treatment is designed to work specifically on KJ.
His doctor at Children's Hospital, Rebecca Ahrens-Nicklas, said it's too soon to know how well the treatment worked, but confirmed KJ is "showing some early signs of benefit."
The potential positives of CRISPR treatment are inspiring. According to team member Dr. Kiran Musunuru, "I don't think I'm exaggerating when I say that this is the future of medicine." He sees this as "the first step towards the use of gene-editing therapies to treat a wide variety of rare genetic disorders for which there are actually very few treatments currently in development at all."
But the team at DARPA is watching these rapid advances with dual uses in mind. Could gene editing be done on all babies born in the U.S. to control populations before they grow into threats? Or even before they are born?
Dr. Musunuru and his team have already been testing gene editing in monkeys before birth. "What we found was astounding," he says. Treating fetuses while they are still in the womb is very much a possibility.
Sources:
https://www.newscientist.com/article/2480365-baby-with-rare-disease-given-world-first-personal-crispr-gene-therapy/
https://www.newscientist.com/article/2403426-sickle-cell-crispr-cure-is-the-start-of-a-revolution-in-medicine/
https://www.newscientist.com/article/mg25834460-200-how-crispr-therapy-could-cure-everything-from-cancer-to-infertility/
https://www.newscientist.com/article/2456378-why-gene-editors-want-to-treat-fetuses-when-they-are-still-in-the-womb/