Canavan Disease

Gene therapy pioneered at UNC is given to children with rare disorder

Published in Gene Therapy Weekly, June 19th, 2003

A form of gene therapy created and produced at the University of North Carolina at Chapel Hill and approved by the U.S. Food and Drug Administration has been given to children with a rare, inherited neurological disorder.

This work involved collaboration with investigators at the Robert Wood Johnson Medical School, of the University of Medicine and Dentistry of New Jersey, and was led by Dr. Paola Leone and a group of 16 investigators and surgeons.

The children have Canavan disease, characterized by spongy degeneration of the brain's white matter. The disease affects the growth of the fatty myelin sheath insulating nerve fibers.

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Source: Gene Therapy Weekly (2003-06-19)

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