Paola Leone, Ph.D., is perhaps the world's leading researcher on Canavan disease. She has received the UNESCO Lifetime Achievement Award for her devotion to the search for a cure for Canavan disease. Doctor of Philosophy (Ph. ... Canavan disease is an inherited disorder that causes progressive damage to nerve cells in the brain. ... UNESCO logo UNESCO (United Nations Educational, Scientific and Cultural Organization) is a specialized agency of the United Nations established in 1945. ...
Leone, Freese and Matthew During, M.D., professor of neurosurgery at Jefferson Medical College, then all at Yale University, were contacted by families with children with Canavan disease to see about the possibility of developing a therapy for the disease.
During, Leone and Freese developed a gene delivery system based on liposomes, or fatty molecules, and polymers, rather than viruses, to deliver the genetic information for the enzyme into the brain.
Several photographs taken by Dr. Leone showing patients, families, doctors and researchers participating in the earlier trial currently are on display at The Genomic Revolution exhibit at the American Museum of Natural History in New York until January 1, 2002.
PaolaLeone from Robert Wood Johnson Medical School in New Jersey is forging ahead with her gene therapy trial in children with Canavan disease.
You will recall that during her stay at Thomas Jefferson University, Dr. PaolaLeone in 2001 had performed gene therapy in three Canavan children, under a protocol involving infusion into the brain of a liquid solution containing several billion particles of an adeno-associated virus.
Leone reports that in the 18 months following surgery, the treated children showed normalization of neurochemistry in the brain and increases in their myelin content.
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