More than 80 percent of rare diseases are believed to have a genetic component. Today, with the first gene therapies approved in the United States, many people affected by rare diseases are beginning to see gene therapy as a real possibility in their lifetimes. Because more than half of rare disease patients are children, these advances are especially meaningful.

To identify the questions that patients and caregivers have about gene therapy, the National Organization for Rare Disorders (NORD) reached out to its members and social media followers. The following are some of the most frequently asked questions.

1. Is gene therapy a cure?

Because it is still in the experimental phase, it is not known if gene therapy is a cure, but that is the goal.

2. Why is gene therapy getting so much attention now?

Recently, there have been significant discoveries about our DNA and how it can be manipulated. Pre-clinical successes have increased, and the FDA has approved some gene therapy drugs. These include a treatment for children and young adults with a form of cancer that progresses quickly called acute lymphoblastic leukemia, and a treatment used to help children and adults with an inherited form of vision loss that may result in blindness.

3. Can the changes /results of gene therapy be passed on to future generations?

Right now, gene therapy is only performed on cells that cannot be passed to offspring. In the future, gene therapies may be developed that could benefit future generations.

4. Why does it cost so much?

At this time, the process for researching and developing gene therapies is expensive, and it is difficult to make progress on a large scale. In addition, companies need to invest in ongoing costs tied to monitoring the drug for several years after it has been administered to patients.

5. How can I find out if a gene therapy clinical trial is available for my condition?

The best way to find out is by asking your specialty care provider or by visiting, where all United States clinical studies are publicly listed.