You can thank CRISPR for that
The takeaway: CRISPR gene editing technology is now a decade old—while we do not yet have FDA-approved CRISPR therapy, there is a lot to celebrate. Let's look at the five big ways it is revolutionizing medicine and more.
CRISPR—the gene editing technology that is opening up new possibilities for the delivery of precision medicine—just turned 10.
The technology has been called a “revolution in progress” and “unmatched and unparalleled” for its ability to location a specific spot in a strand of DNA and cut, add, or trade out different parts of it.
Writing in Science, CRISPR’s co-inventor, biochemist Jennifer Doudna, wrote: “The past decade has witnessed the discovery, engineering, and deployment of RNA-programmed genome editors across many applications. By leveraging CRISPR-Cas9's most fundamental activity to create a targeted genetic disruption in a gene or gene regulatory element, scientists have built successful platforms for the rapid creation of knockout mice and other animal models, genetic screening, and multiplexed editing.”
So just what does this “revolution in progress” look like? Are we on the cusp of CRISPR therapeutics? Let’s look at five of the ways CRISPR is revolutionizing medicine:
- CRISPR is helping to tackle food crises
Growing global populations, transportation bottlenecks, climate change, and strife continue to create concerns about the availability of food. While CRISPR can’t solve any of those problems, it can be used to genetically modify foods to improve their nutritional value, protect them against pests, and even extend their shelf life.
For example, scientists used CRISPR to disable the gene that causes mushrooms to brown when they are cut. Researchers have tinkered with the DNA of the cacao plant to create strains that resist pests. And, of course, CRISPR is helping in the drive to grow meat in a lab, which would reduce the impact that many livestock species have on the environment, from water use to methane emissions.
- CRISPR is leading a revolution in viral detection
CRISPR could be critical aid for people with genetic disorders Down syndrome, cystic fibrosis, and Huntington’s disease. Currently, applications for CRISPR are being explored that would give people better access to medical testing that would detect serious diseases. Using blood, saliva, or urine, it is expected that tests could be developed that would be available to people who want a fast and accurate test for major diseases.
- CRISPR-Cas9 may play a role in curing disease
Are we reaching the long-term goal of CRISPR sickle cell gene therapy? CRISPR-Cas9 applications are increasing. Trials are underway that use CRISPR gene therapy and CRISPR-based therapy to treat diseases such as sickle cell and beta thalassemia. While previous treatment options for these genetic disorders only went after symptoms, CRISPR could actually cure them genetically. For example, CRISPR can treat sickle cell disease by correcting defective genes. CRISPR therapeutics for sickle cell disease have long been a goal of researchers.
Research is pushing the use of CRISPR beyond those ailments and into a whole new set, with CRISPR technology being applied to high cholesterol, HIV, and even muscular dystrophy. Cancer remains the major focus of research, as all cancers are linked to problems in genes. While there are no cures yet, research is focusing on immunotherapy, which uses a body’s immune system to fight cancer. In that effort, CRISPR has been tapped to boost the immune system’s T cells, editing them to spot cancer then kill the cancer cells. In research settings (CART- T immunotherapy), CRISPR has also been used to change the genes in cancer cells and in the process slow down how fast the cancer can spread.
- CRISPR can make drugs better
Drugs are typically tested in animals before being tested in humans, with species such as mice and monkeys being the most popular. While animal models often predict how a drug will perform in humans, that is not always the case. CRISPR is helping to change that by allowing researchers to create more sophisticated cellular models that do a better job of predicting how the drug might work in humans, which would save researchers time and money while also reducing the number of animals used in drug development. Getting CRISPR drugs past through the FDA approval process will be challenging, and CRISPR-derived drugs for rare diseases are likely to be ridiculously expensive.
- CRISPR may help fight climate change
Efforts are underway that would use CRISPR to edit the genomes of plants so they emit less methane and absorb more carbon dioxide. Further, CRISPR is being used to improve the yield of plants and improve the nutritional value of foods while also extending their shelf lives—all actions that would reduce transportation needs and foot wastage and, by extension, greenhouse gas emissions. Meanwhile, the potential to use CRISPR to generate biofuels is also growing. This would reduce greenhouse gas emissions and combat some of the effects of climate change. Finally, CRISPR is being applied to bacteria that would create a process for generating fuel from waste such as methane in landfills.
CRISPR has shown its value by being so powerful. What will the next 10 years hold?
Writing in Science, Doudna predicted even more big breakthroughs.
“In the decade ahead, genome editing research and applications will continue to expand and will intersect with advances in technologies, such as machine learning, live cell imaging, and sequencing.” she wrote. “A combination of discovery and engineering will diversify and refine the CRISPR toolbox to combat current challenges and enable more wide-ranging applications in both fundamental and applied research. Just as during the advent of CRISPR genome editing, a combination of scientific curiosity and the desire to benefit society will drive the next decade of innovation in CRISPR technology.”
What will future applications of CRISPR be? Applications for CRISPR are sure to increase, including those for CRISPR cancer therapy. Whatever it is that happens, IDT will be at the forefront of supporting researchers trying to reach these lofty new heights.
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