Novel AAV serotypes for improved ocular gene transfer

C Lebherz, A Maguire, W Tang… - The Journal of Gene …, 2008 - Wiley Online Library
C Lebherz, A Maguire, W Tang, J Bennett, JM Wilson
The Journal of Gene Medicine: A cross‐disciplinary journal for …, 2008Wiley Online Library
Some of the most successful gene therapy results have been obtained using recombinant
viral vectors to treat animal models of inherited and acquired ocular diseases. Clinical trials
using adenovirus vector systems have been initiated for two ocular diseases. Adeno‐
associated viruses (AAVs) represent an attractive alternative to adenoviral vector systems as
they enable stable and long‐term expression and can target a variety of different ocular cell
types depending on the capsid serotype; recently clinical trails for congenital blindness was …
Abstract
Some of the most successful gene therapy results have been obtained using recombinant viral vectors to treat animal models of inherited and acquired ocular diseases. Clinical trials using adenovirus vector systems have been initiated for two ocular diseases. Adeno‐associated viruses (AAVs) represent an attractive alternative to adenoviral vector systems as they enable stable and long‐term expression and can target a variety of different ocular cell types depending on the capsid serotype; recently clinical trails for congenital blindness was initiated with a vector‐based AAV serotype 2. High levels of retinal gene transfer have been achieved using vectors based on AAV serotypes 1, 2, 4 and 5. This report compares the gene transfer efficacy and stability of expression of vector systems based on three novel AAV serotypes: AAV7, 8, 9, with the established vectors AAV1, 2, 5. We show here that AAV7 and 8 enable superior long‐term transduction of retinal and also anterior chamber structures. Copyright © 2008 John Wiley & Sons, Ltd.
Wiley Online Library