New gene therapy halts 2 boys’ rare brain disease! French scientists mixed gene therapy and bone marrow transplants in two boys to apparently halt a brain disease that can kill by adolescence. The surprise ingredient: They disabled the HIV virus so it couldn’t cause AIDS, and then used it to carry in the healthy new gene.
Lead researcher Dr. Patrick Aubourg of the University Paris-Descartes said the experiment marks the first time researchers have tried that long-contemplated step in people and the first effective gene therapy against a severe brain disease.
According to Dr. Kenneth Cornetta, president of the American Society of Gene and Cell Therapy, although it’s a small, first-step study, it has “exciting implications” for other blood and immune disorders that had been feared beyond gene therapy’s reach.
“This study shows the power of combining gene therapy and cell therapy,” added Cornetta, whose own lab at Indiana University has long researched how to safely develop gene delivery using lentiviruses, HIV’s family.
The research was published in Friday’s edition of the journal Science.
