IN BRIEF
  • This new method could prevent a cancer-causing side effect of gene therapy, which cured 17 of 20 patients of a life-threatening immunodeficiency but caused 5 to develop leukemia.
  • Gene therapy is helping us attack genetic disorders at their sources, and in the future, it could even allow us to reverse blindness or stop aging.

A NEW DELIVERY SYSTEM

Gene therapy is allowing us to attack genetic disorders at their source, giving us a way to replace defective genes with beneficial ones that function the way they are supposed to. To deliver these beneficial genes, doctors essentially replace the “bad” part of a virus with the beneficial gene. By leaving intact the part of the virus that infects the cells of its host, it becomes a delivery system for our new gene. However, the process of delivering these genes to their target cell can produce a nasty byproduct: cancer.

To combat this infrequent but serious side effect of gene therapy, Washington State Universityresearcher Grant Trobridge has developed a new way for viruses to deliver genes to their target cells. Now, he and his team propose using this new method to develop a stem cell gene therapy treatment for SCID-X1, a life-threatening immunodeficiency that affects newborns.

Also known as “Boy in the Bubble Syndrome,” SCID-X1 has been effectively treated using gene therapy before, with 17 of 20 patients in a clinical trial responding to treatment. Unfortunately, five of those patients then developed leukemia. To prevent this, Trobridge and his team have proposed using a foamy retrovirus to deliver the beneficial genes. Unlike other kinds of retroviruses, this type doesn’t typically infect humans and is less likely to activate genes near the target, including those with the potential to cause cancer.

Credit PublicDomainPictures/Pixabay
Credit PublicDomainPictures/Pixabay

A REVOLUTIONARY TREATMENT

“Our goal is to develop a safe and effective therapy for SCID-X patients and their families,” Trobridge said in a press release. “We’ve started to translate this in collaboration with other scientists and medical doctors into the clinic.”

While the new treatment option won’t be ready for clinical trials for another five years or so according to Trobridge, it marks yet another advancement in the growing field of gene therapy. It’s already being used to effectively treat ADA-SKID, create biological pacemakers, and correct genetic mutations. In the future, it could even allow us to reverse blindness or stop aging. The possibilities are truly unlimited if we can harness the power of our own DNA.