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台灣視網膜色素病變協會
:::左側區塊

Gene therapy - great news

發佈日期:2008/05/29 pm07:49:43

內容:
Dear friends,

today and tomorrow are exciting days for all people with inherited retinal degenrations. Two research groups, the group of Prof. Robin Ali from Moorefields Eye Hospital and the group of Prof. jean Bennett from Plhiladelphia University announce the first results for their gene therapy trial in RPE65. Both trials were crowned by success in improving vision and having no major side effects. Both studies are published today in the New England Journal of Medicine and Prof. Bennett addressed today the ARVO community and Prof. Robin Ali will do so tomorrow morning.

Please find enclosed the two press releases by the respective research groups as well as a communication of FFB USA to be posted on their website. Background information as well as the publications are posted unter the member only section of www.retina-international.org.

I kindly ask you to discuss these results with your scientific advisors before communicating to the public.

I welcome enthusiasticly these great results and congratulate the researchers, but also our member organistions, such as FFB USA, BRPS in the UK, Foresight UAE and the individuals having supported this major efforts financially.

With these landmark results proof of principle is given, that gene therapy in autosomal-recessive retinal eye disease is possible. Now we will have to work hard, to find all individuals with LCA world-wide and to genotype all patients with inherited retinal diseases. We will have to focus to those diseases, that might be suitable candidates in the near future, such as Stargardt disease, Achromatopsia, Bardet Biedl Syndrome etc. To do so, patient registries are extremely important and I call on all our member organisations to do their best to promote those registers in their own country.

With kind regards