At PatientWorthy, we’ve talked a lot about gene therapy, but we’ve never really taken the time to explain it. What the heck is gene therapy? How does it work?
Well, like your relationship with pharmaceutical companies, it’s complicated. So, let’s aim for simplicity.
Gene therapy, according to Genetic Literacy Project, is when a patient’s stem cells are surgically removed, altered and combined with corrected versions of the original defective gene, and then, essentially, put back into the patient. Easy, enough.
Don’t confuse gene therapy with stem-cell transplants–they are very different, the latter involving a donor and a host.
In recent years, a lot of research has gone into gene therapy, for conditions like beta-thalassemia, primary immune deficiency diseases such as severe combined immunodeficiency (SCID), and Wiskott-Aldrich syndrome.
Thanks to gene therapy, people with chronic disease can look to their futures a bit more hopeful.
And there you have it. Gene therapy in a nut-shell. Now you can impress all your friends at parties with your new-found knowledge.
Read more about gene therapy here.