The whole world is abuzz about CRISPR, the new technology that’s allowing scientists to easily edit genetic data. This development is poised to fundamentally change our relationship with genetic disease. In the future, we may be able to do more than treat the symptoms of genetic disease — instead, we might strike directly at the DNA causing maladies such as “bubble boy” syndrome, muscular dystrophy and sickle cell disease.
But the biggest impact CRISPR will have on most people’s lives won’t be curing genetic diseases. It’s much larger: the widening of our horizon of discovery, which could lead to advances we can’t even imagine.
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CRISPR stands for “clustered regularly interspaced short palindromic repeats.” The obtuse name refers to the discoveries that enabled its development as a gene editing tool. Scientists first noticed in the late 1990s and early 2000s that many bacteria hold little bits of viral DNA (CRISPRs) within their own genomes. After years of intense research — work that was unrelated to gene editing — it was finally understood that these bits of DNA form part of an immune system that allows bacteria to fight off viruses.
Specialized proteins form part of the bacterial immune system and use these bits of viral DNA to recognize and cut the genomes of invading viruses, protecting the bacteria from viral attack. By developing these proteins into easily programmable scissors that can recognize and cut other DNA, CRISPR became a hugely powerful tool for editing genomes for other purposes.
CRISPR represents a triumph fundamental to research: Undirected scientific curiosity can lead to unexpected breakthroughs that improve our lives. CRISPR is able to dramatically accelerate biological discovery by “democratizing” gene editing. The tool gives scientists the ability to make new insights into the workings of life, for example, by testing how genes function during health and disease. The application of similarly fundamental biological discoveries has formed the cornerstone of almost every advance in human health, from new cancer drugs to cutting-edge cholesterol therapies.
Gene editing technology has been around for a while, but earlier tools required relatively back-breaking effort, so few researchers did those kinds of experiments. CRISPR-based editing is fast and easy, and it’s now being used in all sorts of contexts to ask myriad questions in all realms of biology. This happens in settings with readily recognizable and easily applicable real-world benefits, such as human cells or agricultural crops, but research is also being done for the purpose of pure discovery on fish, field mice, butterflies and tiny crabs.
Today, using CRISPR, we can make changes to genomic DNA and better understand what each element of the genome does. And what if some of that data leads to the next big unanticipated breakthrough? What if the field mouse gives us clues to treat neurological disorders? What if the tiny crab holds the key to the regeneration of severed limbs? We can’t know until we look, and CRISPR gives us the means to go looking.
This opportunity to dive deep into biological systems that were previously impenetrable is the real power of CRISPR. And scientists realize it. By my last count, there are three new peer-reviewed publications that mention using CRISPR per day. And that number is only increasing.
It’s staggering to imagine all of the amazing, completely unanticipated things that democratized gene editing will enable us to discover in the future. And as a new generation of scientists is trained with this kind of technology in easy reach, they’ll come up with even more creative uses and unearth more groundbreaking questions. CRISPR will change our relationship with genetic disease, but its ability to accelerate fundamental research also lets us learn much more about the biological world in which we live.
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