Message from David Brint, President of FRR

Dear Friends:
As some of you may have heard, Jean Bennett at the Children's Hospital of Philadelphia has announced the initial results of the RPE 65 gene therapy trial. Jean has reported that all three of the injected patients have had some restored vision. This includes vision of up to three lines on an eye chart andimproved ambulatory vision.
This is the most significant result for the restoration of sight in history.
You all should know that the current trial is only a safety trial and has only included a half dose of the gene. What this all means is that the gene vectoris not causing any health problems to date and that the results may improve with increased dosing.
Gene therapy will be a potential treatment for all forms of LCA where photoreceptors are intact.
The FRR has been a supporter of Jean Bennett's work and we are proud of her team's accomplishment.
The attached web links will give you more information
http://abcnews.go.com/Health/wireStory?id=4737873http://content.nejm.org/cgi/content/full/NEJMoa0802268
Sincerely,
David Brint
President FRR