WEDNESDAY, Dec. 19 (HealthDay News) -- Gene therapy could offer
a safe option for treating children with a rare degenerative
brain disease, a preliminary study suggests. The condition,
known as Canavan disease, is caused by a mutation in the ASPA
gene, which codes for an enzyme called aspartoacylase. Without
that enzyme, a chemical known as N-acetylaspartic acid (NAA)
builds up in the brain and prevents nerve cells from developing
their normal protective sheath. Eventually, the brain
degenerates into spongy tissue with fluid-filled spaces. Canavan
disease is rare, with an estimated 600 cases worldwide,
according to Paola Leone, the lead researcher on the new study
and an associate professor of cell biology at the University of
Medicine and Dentistry of New Jersey in Stratford, N.J. Both
parents have to carry the mutated gene for a child to be
affected, and the disease is most common among people of
Ashkenazi Jewish heritage. Babies born with Canavan disease
never develop normal movement and thinking abilities