medicine, stem cells, technology

What Has Gene Therapy Done For You Lately?

“Gene therapy” might sound like science fiction, but the field has actually made real, tangible progress over the last few years. Gene therapy refers to using DNA to treat a disease or condition (usually a genetic one). Most often, the DNA specifically goes into a patient and replaces a non-functional gene with a working one.

blood cells
This is a scanning electron microscope image of normal circulating human blood. Blood stem cells have been modified through gene therapy to treat people with Wiskott-Aldrich Syndrome. (Image credit: Bruce Wetzel and Harry Schaefer)

Here are a few recent examples of how gene therapy has been used:

  • In mid-2013, researchers announced they’d developed a better gene therapy approach for treating Wiskott-Aldrich Syndrome. The Syndrome is an inherited immune system disorder that’s due to a mutated gene. It can be treated by genetically modifying a patient’s own blood stem cells to have a fixed copy of the gene (specifically by removing the patient’s stem cells, using a virus in a laboratory to deliver the corrected gene to those cells, and then putting them back into the patient). Treatments are also done using donor bone marrow or stem cells, but it can be hard to find a good matching donor.
  • In late 2012, the results of a 10-year study using gene therapy to treat patients with severe coronary artery disease showed that there’s promise for such a strategy. Specifically, some patients received injections of an angiogenic growth factor gene (VEGF) directly into their heart muscles to help them rebuild their damaged blood vessels. These patients had improved outcomes compared to patients who didn’t have the gene therapy.
  • Gene therapies have been explored for many other diseases and conditions, and include potential treatments for Duchenne Muscular Dystrophy, some forms of blindness, and even regenerative surgery.

And techniques are improving all the time, further expanding the possible applications of gene therapy. Just recently, there have been reports of using magnetic nanoparticles and lipid nanoparticles to better deliver genes. And with an improved understanding of viruses, scientists may be able to distribute more genetic material to a larger part of a patient’s body, which has been a problem in some cases. Other hurdles remain to be overcome for gene therapy to be more widely used, but progress is certainly being made.

So while we don’t have flying cars or teleporting transporters yet, gene therapy is one technology that has been making its way out of science fiction and into patients who need it.


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