For a few lucky patients, 2016 was the year when gene therapy turned from promises to cures.
So what is gene therapy, anyway? The U.S. Food and Drug Administration says it’s any treatment in which a replacement gene is added to a person’s body or a disease-causing one is inactivated.
We started the year with the tale of Glybera, heralded as the first gene treatment ever approved that sought to correct an inherited gene error.
The first out-and-out-cure: The dream of gene therapy is to fix your DNA so you’re not sick anymore-a “Cure.” During 2016, Italian scientists at Milan’s San Raffaele Telethon Institute for Gene Therapy reported that they had cured 18 children of a rare but terrible immune deficiency disease, ADA-SCID. They removed the children’s bone marrow, added a gene to make the ADA enzyme their bodies lack, and replaced it.
Gene editing: Today’s gene therapy is about adding genes-say, to replace one in your body that isn’t working. What if you need to delete a misbehaving gene, or want to actually rewrite it? To do that, you’ll want to do some gene editing.
U.S. approvals next year: With so many promising results in human tests, 2017 will be the year that several gene therapies end up before the Food and Drug Administration.