UNC study may improve gene ther...( n this database Samulski said. We're...)

UNC study may improve gene therapy safety

n this database," Samulski said. "We're laying the foundation here for that to happen."

Samulski, a pioneer in AAV research, said the new study further confirms his choice to study and develop altered AAV for gene therapy. Along with its potential for fewer toxic effects than that of many other viruses studied for use in gene therapy, a gene delivered via AAV remains active in cells for months or even years, he added.

Last year, a form of gene therapy created and developed in Samulski's laboratory and based on AAV was approved by the U.S. Food and Drug Administration and given to children with Canavan disease, a rare, inherited neurological disorder. It marked the first FDA approval for clinical use of an AAV vector for gene therapy that was produced by a U.S. academic institution.

'"/>

Contact: Leslie H. Lang
llang@med.unc.edu
919-843-9687
University of North Carolina School of Medicine
23-Feb-2004

Page: 1 2 3
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