Minute to Understanding January 11, 2019

What is gene therapy?

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.

If you had a hole in your favorite pair of blue jeans, your tailor could fix them by simply patching them up with new fabric.

But if something goes wrong with one of the 24,000 genes in your body? Your doctor’s approach to repairing them is not so simple.  

Unfortunately, mutations in our genes can cause life-threatening diseases. And sometimes there is no drug or surgery available for your doctor to prescribe.

Wouldn’t the best strategy for a permanent repair be to patch up the mutated gene, or at least provide a correct copy to cells in the body? This is the hope of gene therapy vs. hit-or-miss drug therapies.

Many studies in animal models have been successful and offer promise for treating people. But first, scientists need to make sure of the long-term effects of gene therapy before the technique can be adapted for human use.

We think it’s a solution worth exploring because gene therapy could not only cure a variety of different genetic diseases, but it could also give a big kick in the “pants” to cancer.