Vascular-targeted gene therapies,which have the potential to treat many of the leading causes of mortality in the western world, have been ineffective due to poor vascular gene transfer. An alternative is the use of virus serotypes and the incorporation of vascular targeting ligands into vectors to increase vascular gene transfer.
Adeno-associated virus (AAV) 1 has shown the most promise among the AAV vectors for the transduction of vascular endothelial cells. As detailed in research published in Gene Therapy (Vol. 13, p. 926-931), the authors (M D Stachler and J S Bartlett) combined two AAV1 capsid protein modifications to enhance vascular gene transfer and allow easy purification of vector particles. Their results suggest that mosaic virions hold significant promise for targeted gene delivery to the vasculature.
Dr JS Bartlett, Gene Therapy Center, Columbus Children's Research Institute, 700 Children's Drive, Columbus, OH 43205, USA. E-mail: firstname.lastname@example.org
Abstract available online.
(C) Gene Therapy.
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