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  • Writer's pictureSinead Mackintosh

Gene Therapy

Since the whole world is feeling like a bad sci-fi movie, let’s talk about something else that sounds like something out of a mad scientist’s lab: gene therapy. As the name implies, it is a medical treatment involving genetics, but what can it (or could it) actually do?

Gene therapy was created to insert genetic material into cells to “fix” abnormal genes or make a beneficial protein that the cell/organism needs. This genetic material needs a way of getting into the cell and being useful. Scientists use a “vector” to do just that. The vectors usually are viruses, and I know that right now, viruses seem terrible, but they can be quite useful. These vector viruses are modified so that they do not cause disease when used in humans. There are several different types of viruses, and they are suited to various tasks. However, the main challenge is getting the genetic information into the vector and then to be useful inside of the cell.

This is a problem because sometimes the genetic material is too large to fit in the vector. Also, sometimes the body thinks that it is a dangerous intruder and destroys the genetic material before it has a chance to be therapeutic. However, numerous ongoing clinical trials are trying to overcome these and other barriers. There are a couple of FDA approved gene therapies such as Zolgensma that are used to treat children under the age of 2 who have spinal muscular atrophy with a specific type of genetic mutation. This drug was only approved last year in May. So it has a lot of potential to help families with affected children or who are at risk of having affected children.

Gene therapy could be used to treat cancers, heart diseases, diabetes, HIV/AIDS, hemophilia, and cystic fibrosis, to name a few. So it’s easy to see why this is such an appealing line of treatment and research. But it is not there yet and still has a long road ahead before large scale treatments are accessible.

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