News Health and Medicine Gene therapy cocktail shows promise in long-term clinical trial for rare fatal brain disorder
| Gene therapy cocktail shows promise in long-term clinical trial for rare fatal brain disorder |
|
| Wednesday, December 19, 2012 | |
|
Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder. A report of the trial appears in the Dec. 19, 2012 online edition of the journal Science Translational Medicine. For full release |