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Top Story

New 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. National Science Foundation-funded 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. Only a few gene therapies have been approved for human use so far, but scientists are working on and testing new gene therapy treatments for diseases such as sickle cell anemia, hemophilia and congenital eye disease, among many others. 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.

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