Jefferson researchers use gene therapy for rare, inherited brain disease
A team of scientists, neurosurgeons and physicians at Thomas Jefferson University Hospital are for the first time using a novel form of gene therapy for Canavan disease, a fatal, neurodegenerative childhood disorder. Armed with a new $2.1 million, three-year NIH grant, researchers recently introduced 90 billion copies of a healthy gene into the brains of three children. Their brains lacked the gene, which makes an enzyme that breaks down an acid substance in the brain.
National Institute of Neurological Disease and Stroke
Thomas Jefferson University
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