![]() Instead, Carlon is exploring a cystic-fibrosis treatment that harnesses a method called base editing, which can change individual DNA letters, or bases - converting an A to a G, for example, or a C to a T. Got mutation? ‘Base editors’ fix genomes one nucleotide at a time But for that, classical CRISPR–Cas9 approaches are of little use: “CRISPR is much better at destroying things than it is at fixing things,” says Gottesdiener. Genome editing offers an opportunity to correct the mutations that cause cystic fibrosis, which affects the lungs and digestive system. Here, Nature looks at the next generation of CRISPR techniques. The regulatory approval of classical CRISPR–Cas9 “sets the stage” for the next generation of genome-editing techniques, says Marianne Carlon, a lung disease specialist at the Laboratory of Respiratory Diseases and Thoracic Surgery at the Catholic University of Leuven in Belgium. And they can make changes, such as switching on genes, that the initial tools couldn’t. These systems edit DNA with more precision and versatility than the original genome editors could achieve. Now, however, there’s a fresh crop of CRISPR-based systems that overcome those limitations. “They can do some remarkable things, but they’re fairly limited.” “We tend to call these the first generation of genome editing,” says Keith Gottesdiener, chief executive officer of Prime Medicine, a company in Cambridge, Massachusetts, that is developing genome-editing therapies. A host of other CRISPR–Cas9 therapies that work on the same principle are in clinical trials as treatments for a range of diseases.Īs sophisticated as these therapies are, they are only the beginning. The therapy, which UK regulators approved on 16 November, disables a gene as a means of treating a genetic blood disorder called sickle cell disease. Less than a month after the world’s first approval of a CRISPR–Cas9 genome-editing therapy, researchers are hoping that the therapy will win its second authorization this week - this time from the United States, with its famously stringent regulators and lucrative health-care market. Update: On 8 December, the US Food and Drug Administration approved the CRISPR–Cas 9 therapy for sickle cell disease described in this story. Credit: Biolution GmbH/Science Photo Library ![]() More versatile genome editors are supplanting the CRISPR-Cas9 editing system (artist’s impression) for experimental treatments.
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