Neurometabolic Diseases Lab

  • Increase font size
  • Default font size
  • Decrease font size
Home On the News
On the News

Gene Therapy Halts Fatal Brain Disease

E-mail Print PDF

From Wired Science
November 5, 2009
By Brandon Keim

Scientists have used gene therapy to halt the progression of adrenoleukodystrophy, a fatal neurodegenerative disease caused by a single defective gene, in two seven-year-old boys.

It took more than a decade to refine the therapy, in which stem cells taken from the boys’ bone marrow were hacked with healthy copies of the gene, then returned to their bodies. Without them, the boys would soon be dead.

Last Updated on Tuesday, 10 November 2009 12:02 Read more...
 


Page 5 of 5

View My Stats