Great news!
Phase 1 gene therapy clinical trials for Leber's Congenital Amaurosis caused by RPE65 have begun at the University of Pennsylvania and the University of Florida! One volunteer patient has received an injection of genes into the retina of one eye. The injection was performed by Shalesh Kaushal, MD, PhD, assistant
professor of ophthalmology at the University of Florida. Six adults and three children between the ages of 8 and 17 years are scheduled to undergo the procedure at the university over the next year.
This is the second gene therapy trial for RPE65, commencing only a few months after the one currently underway at London’s Moorfields Hospital. It will take a while before results from both trials are disclosed, however if things go well the good news should start to leak out soon.
Tuesday, November 20, 2007
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10 comments:
How soon is soon? Are there any updates?
I wish I knew how soon! Be sure that I'll post any updates as soon as I hear more.
Hi, My name is Billy from South Carolina. The one you had mentioned that is in the trial down in Florida was I. I will be glad to answer any questions you may have.
I have had the AAV2RPE65 transfer on Oct 28th 2007 at Shand's Hospital in Gainesville, FL. There is realy no major positive news from me. I had 20/200 vision in my left eye which is the injected eye. I use to be able to read, but at this time I cannot read with it and my vision is quite worse. However, I am starting to notice brighter flashes of light that seem to have something todo with the injection. So, it may take some time to tell if my eye heals to where I am back to my previous state or better than ever. Feel free to e-mail me at irwin.billy@gmail.com
Billy, thank you soooo much for posting! It's wonderful to hear from someone who is really doing it. Then, it's real!
Expect lots of questions now....:-)
Thanks again
Fran
There is another trial at Children's Hospital of Philadelphia which started earlier this fall--- there is announcement about it posted on the FRR website.
Hi and thanks so much for the information! I checked the FRR site, and I could only find reference to the Philadelphia trial in the October newsletter, which I also published on this blog earlier. Is there anything new I can't find?? If so, please let us know. I haven't posted anything on this trial because I have no information, except that it was supposed to begin in the fall. I hope it has begun!
Thanks again
Fran
Thanks so much for creating this blog!
Thank Billy for sharing your participation.
I join the others in being anxious to continue to hear of the results.
Kim, thank you for leaving your comment! Please check today's updates to this story. This is an incredibly exciting day for research!!
Fran
Please update us, my daughter is considering the trial as well, the results of her genetic tests are due by end of year, would like any/all input you may have.
Hello,
be sure I will update this blog regularly with any news. An article has just been published confirming the excelent short-term results of the RPE65 trials. I hope your daughter's genetic testing reveals she has the RPE65 mutation, however gene therapy trials for other LCA genes are not too far off in the future.
Thanks for visiting!
Fran
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