Sometimes biology is cruel. Sometimes simply a one-letter change in the human genetic code is the difference between health and a deadly disease. But even though doctors and scientists have long studied disorders caused by these tiny changes, replicating them to study in human stem cells has proven challenging. But now, scientists at the Gladstone Institutes have found a way to efficiently edit the human genome one letter at a time—not only boosting researchers' ability to model human disease, but also paving the way for therapies that cure disease by fixing these so-called 'bugs' in a patient's genetic code.
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