Gene Therapy Extends Lives of Children With Brain Disease

Children born with a rare, genetic brain disorder that causes severe atrophy and often leads to death within three years, are still alive 7 to 10 years after being treated with an experimental gene therapy, a study showed.

The findings, published today in the journal Science Translational Medicine, described the procedure of inserting a virus containing healthy genes into the children’s brains through holes drilled into their skulls. The 13 children, the youngest of whom was 3 months old and diagnosed with the disorder while in the womb, were treated at the Cell and Gene Therapy Center at the University of Medicine and Dentistry of New Jersey in Stratford.