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Matthew Hirsch; Design, Validation, and Translation of AAV Gene Therapy Vectors for the Treatment of Ocular Diseases. Invest. Ophthalmol. Vis. Sci. 201657(12):.
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© ARVO (1962-2015); The Authors (2016-present)
Presentation Description :
This presentation intends to provide an overview of the bench to bedside journey using adeno-associated virus (AAV) vectors for the treatment of genetic diseases with a particular focus on ocular abnormalities. Topics will include the design of AAV transcriptional cassettes, the choice of AAV capsids for specific tissue/cell transduction when administered by different routes, and other emerging trends towards safe and efficient AAV-mediated gene delivery in the eye. Next, validation of the chosen reagents will be addressed including efficacy and toxicity experiments necessary for FDA approval and other ethical obligations. Finally, a brief overview of negotiating the regulatory obstacles will be discussed. Throughout this presentation, our experience with these aspects for the development of AAV gene therapy for a corneal disease will used as an example to illustrate/highlight the considerations of each step in the process.
This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.
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