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Gene Therapy Shows Promise for Rare Children's Brain Disorder
Date:12/19/2012

By Amy Norton
HealthDay Reporter

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. They have weak and stiff muscles, suffer seizures, and have problems eating and even supporting their head. (The disorder causes the head to become unusually large.)

Some children with Canavan disease live into their teens or 20s, but most die before their 10th birthday.

"Fifteen years ago, there was no hope for these children," Leone said.

Back in 2001, though, U.S. regulators gave researchers the go-ahead to study a gene therapy method developed by scientists at the University of North Carolina School of Medicine.

The tactic involved inserting a normal copy of the ASPA gene into a non-infectious virus. That gene-carrying virus, or vector, was then infused directly into the brains of children with Canavan disease, in an attempt to override the defective gene.

Over the course of a few years, the researchers treated 13 children in all, and
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