This RNA-based technique could make gene therapy more effective
Delivering functional genes into cells to replace mutated genes, an approach known as gene therapy, holds potential for treating many types of diseases. The earliest efforts to deliver genes to diseased cells focused on DNA, but many scientists are now exploring the possibility of using RNA instead, which could offer improved safety and easier delivery. MIT biological engineers have now devised a way to regulate the expression of RNA once it gets into cells, giving them precise control over the dose of protein that a patient receives. This technology could allow doctors to more accurately tailor treatment for individual patients, and it also offers a way to quickly turn the genes off, if necessary. “We can control very discretely how different genes are expressed,” says Jacob Becraft, an MIT graduate student and one of the lead authors of the study, which appears in the Oct. 16 issue of Nature Chemical Biology. “Historically,...