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Jefferson Researchers Use Gene Therapy To Treat Rare, Inherited Brain Disease

Scientists Are Hoping The Treatment Provides Some Relief For A Young Girl, And Provides Insights To Future Uses Of Gene Therapy For Other Illnesses

Researchers at Jefferson Medical College are for the first time attempting to use gene therapy to treat Canavan disease, a rare, fatal metabolic brain disorder.

A team of neurosurgeons are treating a four-year-old Illinois girl at Thomas Jefferson University Hospital, Thomas Jefferson University, Philadelphia. The group includes Andrew Freese, M.D., Ph.D., associate professor of neurosurgery and director of neurosurgery research, Matthew During, M.D., professor of neurosurgery, Paola Leone, Ph.D., adjunct assistant professor of neurosurgery, and Giancarlo Barolat, M.D., professor of neurosurgery, as well as their colleagues there and at Yale University.

Canavan disease is an inherited neurological disorder characterized by spongy degeneration of the brain. It primarily affects children of Eastern European or Ashkenazi Jewish background. It is one of a group of genetic disorders called leukodystrophies that affect the growth of the myelin sheath of the nerve fibers in the brain. The myelin sheath is the fatty covering surrounding the nerve cells that acts as an insulator. The disease is caused by a genetic flaw in which an enzyme fails to be produced. The disease is incurable, resulting in the over-production of a toxic compound in the brain, N-acetyl-aspartate (NAA). Death usually occurs between the ages of 5 and 7.

Symptoms of Canavan disease, which appear in early infancy and progress quickly, may include mental retardation, loss of previously acquired motor skills, difficulty feeding, abnormal muscle tone, poor head control, and an abnormally enlarged head. As time progresses, sufferers also become paralyzed, blind, and lose hearing and their interaction with the outside world.

"Children fail to meet normal developmental milestones," Dr. Freese explain
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Contact: Steve Benowitz
steven.benowitz@mail.tju.edu
215-955-6300
Thomas Jefferson University
18-Mar-1998


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