You may have heard the buzz words “gene therapy” without really understanding what it is, or what’s at stake. Gene therapy is when a healthy copy of a gene replaces one that is mutated in some way. And… it’s the introduction of a healthy gene to help the patient fight disease. And… it’s the deactivation of a faulty gene—also called a knockout. It’s lots of things.
About 25 years ago, scientists began to make important inroads with gene therapy, but for every success, there was a set-back.
That’s why international collaboration has been so important. Toward that end, Children’s Hospital in Boston, Massachusetts, became a founding member of the Transatlantic Gene Therapy Consortium (TAGTC). This group of scientists are focused on advancing gene therapy to treat diseases in humans.
A trial now beginning in London will address gene therapy to treat severe combined immunodeficiency, or SCID.
Back in the 1980s, SCID became a more well-known disease because of media coverage concerning a boy named David Vetter, a.k.a “the boy in the bubble.”
After undergoing an unsuccessful bone marrow transplant, David passed away at the age of 14.
