Minute to Understanding: What is gene therapy?

Mutations in our genes can cause life-threatening diseases, and sometimes there is no drug or surgery available for your doctor to prescribe. Enter gene therapy.

Let's talk about your jeans, the jeans you wear! If you had a hole in your favorite pair, you could fix it by patching it up with new fabric. And if you didn't, you might have a little problem.

Now, let's talk about your other genes, the DNA that encodes all of the proteins in your body. Unfortunately, mutations in those genes can cause life-threatening diseases, and sometimes there is no drug or surgery available for treatment. Wouldn't the best strategy be to patch up the mutated gene, or at least deliver a correct copy to cells in the body?

That is the hope of gene therapy, versus say, drug therapy. Studies in animal models are very promising, but scientists need to be sure of the long-term safety of correcting or delivering genes in humans. It's a problem worth solving, because gene therapy could not only cure genetic disease, it could be a real kick in the pants to cancer.