|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.
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