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Gene Therapy

New gene therapy research from University of Pennsylvania, Division of Transfusion Medicine discussed

Published in Gene Therapy Weekly, January 7th, 2010

Research findings, 'Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints,' are discussed in a new report. According to recent research published in the journal Gene Therapy, "Vectors based on the adeno-associated virus (AAV) are attractive and versatile vehicles for in vivo gene transfer. The virus capsid is the primary interface with the cell that defines many pharmacological, immunological and molecular properties."

"Determinants of these interactions are often restricted to a limited number of capsid amino acids. In this study, a portfolio of novel AAV vectors was developed after a...

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