What Is Gene Therapy?

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Gene therapy is typically done for diseases in which there are no other treatment options. Find out how gene therapy is usually used in clinical trials with help from an assistant professor of biology in this free video on gene therapy.

Part of the Video Series: Genetics
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Video Transcript

People who participate in gene therapy are in clinical trials. And typically it's only done for diseases in which there's no other treatment option. So people have no choice except to try the gene therapy for that disease. So it's very early stage as far as treating in disease at this point. If we understand what's gone wrong, precisely, then perhaps we can add back a normal copy of something and fix that problem. In only a few cases though do we actually understand the things that have gone wrong in a particular, if a person has a disease, well enough to put the functional missing gene back. We have a couple of examples of that where a specific enzyme is missing and the person is born with a defective enzyme gene and therefore they don't have it. And it's critical to life. Then if you add back the gene for that enzyme their bodies now can produce it. Engineering a complex organism like a person, which is when you talk about gene therapy we normally talk about human gene engineering. That is even harder than engineering test or model organisms. So we have had, as you would usually have with any kind of therapy, lots of things to learn about how to make the gene actually stay in the body. How to make it express the enzyme that's needed. That doesn't always work correctly. How to make just the right amount, not too much, not too little. And then how to also not have that, inserting that gene, have unexpected effects. So that was the big surprise with some of the early gene therapy. Is that putting the gene into the body had an unanticipated effect. So it caused additional trouble we hadn't predicted.


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