Submitted for Publication: June 29, 2011; final revision received July 23, 2011; accepted July 29, 2011.
Author Contributions: Dr Jacobson had full access to all the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis. Drs Jacobson and Hauswirth contributed equally to the planning and conduct of this clinical trial.
Financial Disclosure: Drs Byrne and Hauswirth and the University of Florida have a financial interest in the use of AAV therapies and own equity in a company (AGTC Inc) that might, in the future, commercialize some aspects of this work. The University of Pennsylvania, the University of Florida, and Cornell University hold a patent on the described gene therapy technology (US Patent 20070077228, “Method for Treating or Retarding the Development of Blindness”).
Funding/Support: The clinical trial was supported by the National Eye Institute (National Institutes of Health, Department of Health and Human Services) grant U10 EY017280.
Additional Information: The Data Safety Monitoring Committee approved this article.
Additional Contributions: We thank the members of the Data and Safety Monitoring Committee: Maryann Redford, DDS, MPH (Program Director, Collaborative Clinical Research, National Eye Institute, National Institutes of Health); Stephen Gange, PhD (Chair); Karl Csaky, MD, PhD; Herbert Goldstein, PhD, PA; Donald Hood, PhD; Ruth Macklin, PhD; Jerry Mendell, MD; Daniel Salomon, MD; and Donald Zack, MD, PhD. We are grateful for the expertise and all the kindness shown to our patients by members of the University of Florida Clinical and Translational Science Institute (General Clinical Research Center) during the early postoperative period.