I always find it exciting when I see an article on gene therapy in The Economist.
Yes, they like to report on new technology early, but they are also not going to risk their reputation on reporting unpromising studies or research.
The article, Gene Therapy: Ingenious, fixing a body's broken genes is becoming possible, published this week, is a general gene therapy article but the first two success cases mentioned are both related to blindness. They don't reference any Achromatopsia gene therapy, but report positive results for choroideremia and Leber’s congenital amaurosis. Additionally, both studies appear to have the same delivery mechanism as would be used for Achromatopsia, although the article is not specific on method used in the first case. (I also like the picture accompanying the article: clever and cute.)
I always feel a surge of optimism when I read these types of articles, I start to believe that there is hope that Charlotte will be a recipient of treatment. I don't know when, or how what difference it might make but I like to know that there are people out there working on this stuff and there is money being found to pay for it. All of my previous hesitation around such a new technology still applies but it is nice to have a little boost once in a while.
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Hi Cathy- my 4 year old daughter has A, and we just went to the Achromatopsia conference in Florida. Gene therapy is definitely on the horizon - the estimate was that something would be approved by the FDA here in the US in 5-6 years, which seems crazy fast. I think Moorfield Eye Hospital is also starting to do the same kind of work there. If your daughter hasn't had genetic testing yet, it might be worthwhile. There is also a terrific book called The Forever Fix that does a great job explaining genetic therapy to laypeople, and it focuses on the LCA2 trials at University of Pennsylvania.
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