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When the guide RNA matches up with the target gene's DNA, Cas cuts the DNA. The guide RNA partners with Cas and-true to its name-leads Cas to the target. Scientists design the guide RNA to mirror the DNA of the gene to be edited (called the target). In the laboratory, the CRISPR tool consists of two main actors: a guide RNA and a DNA-cutting enzyme, most commonly one called Cas9. Within a handful of years, multiple groups had successfully adapted the system to edit virtually any section of DNA, first in the cells of other microbes, and then eventually in human cells.Ĭredit: National Institute of General Medical Sciences, National Institutes of Health If the same germ tries to attack again, those DNA segments (turned into short pieces of RNA) help an enzyme called Cas find and slice up the invader’s DNA.Īfter this defense system was discovered, scientists realized that it had the makings of a versatile gene-editing tool. To protect themselves against invaders like viruses, these microbes capture snippets of the intruder’s DNA and store them away as segments called CRISPRs, or clustered regularly interspersed short palindromic repeats. In this case, the idea was borrowed from a simple defense mechanism found in some microbes, such as bacteria. Like many other advances in science and medicine, CRISPR was inspired by nature. In a small study, for example, researchers tested a cancer treatment involving immune cells that were CRISPR-edited to better hunt down and attack cancer.ĭespite all the excitement, scientists have been proceeding cautiously, feeling out the tool’s strengths and pitfalls, setting best practices, and debating the social and ethical consequences of gene editing in humans. Now CRISPR is moving out of lab dishes and into trials of people with cancer. “CRISPR is becoming a mainstream methodology used in many cancer biology studies because of the convenience of the technique,” said Jerry Li, M.D., Ph.D., of NCI’s Division of Cancer Biology. As soon as CRISPR made its way onto the shelves and freezers of labs around the world, cancer researchers jumped at the chance to use it. The new tool has taken the research world by storm, markedly shifting the line between possible and impossible. Although several methods of gene editing have been developed over the years, none has really fit the bill for a quick, easy, and cheap technology.īut a game-changer occurred in 2013, when several researchers showed that a gene-editing tool called CRISPR could alter the DNA of human cells like a very precise and easy-to-use pair of scissors. Credit: Ernesto del Aguila III, National Human Genome Research InstituteĮver since scientists realized that changes in DNA cause cancer, they have been searching for an easy way to correct those changes by manipulating DNA.