Pre‐existing immunity to adeno‐associated virus (AAV)2 limits transgene expression following intracerebral AAV2‐based gene delivery in a 6‐hydroxydopamine model of Parkinson's disease

ConclusionsOur findings indicate that intracerebral AAV2‐based gene therapy is compromised in rats with pre‐existing immunity to AAV2. By contrast, a local neuroinflammatory response, caused by intrastriatal a 6‐OHDA injection, does not affect viral vector‐mediated transgene expression. Our results emphasize the importance of monitoring circulating AAV‐specific neutralizing antibodies in patients undergoing intracerebral gene therapy using AAV vectors. Copyright © 2014 John Wiley & Sons, Ltd.
Source: The Journal of Gene Medicine - Category: Genetics & Stem Cells Authors: Tags: Research Article Source Type: research