Gene therapy may boost life expectancy in rare brain disorder

12/19/2012 | Bloomberg Businessweek · U.S. News & World Report

An experimental gene therapy in which healthy genes were inserted into patients' brain cells via a virus to produce the enzyme aspartoacylase extended the lives of children with a rare brain disorder called Canavan disease, a study showed. The treatment slowed down the degeneration of brain tissues and improved the quality of life of the children with fewer seizures, better sleep quality, and more mobility and alertness. The findings appear in the journal Science Translational Medicine.

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Bloomberg Businessweek · U.S. News & World Report

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