Gene Therapy Cocktail Shows Promise for Rare Fatal Brain Disorder

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.

The form of gene therapy was created and developed at the University of North Carolina School of Medicine. The work was spearheaded by R. Jude Samulski, PhD, a study senior author, professor of pharmacology and director of UNC’s Gene Therapy Center. The treatment uses a virus (adeno-associated virus, or AAV) as a “viral vector” meticulously tailored to enter the brain and safely switch good genes for bad. For more information, click here to read the full release.