Gene therapy of eye diseases. Ataluren for aniridia. Luxturna for retinal dystrophy. 12

Gene therapy of eye diseases. Ataluren for aniridia. Luxturna for retinal dystrophy. 12

Gene therapy of eye diseases. Ataluren for aniridia. Luxturna for retinal dystrophy. 12

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Gene therapy of eye diseases. What is the future in gene therapy for eye diseases? And where is the most progress in gene therapy being made today in ophthalmology? So gene therapy is a challenge today. We have so many, of course, rare eye diseases. But the interest in eye disease is that an eye is a small lab for medical research. And so, the first pharmaceutical medication is just about to be approved. It is approved now. It was approved in August for gene therapy in ocular disease in retinal disease. This is Luxturna for RPE65 mutation in children with retinal dystrophy. So, the concept is very interesting because we go with a viral carrier and a protein that can be replicated inside the retina. And so we go under the eye, and we also go under the retina to put the viral carrier agent and to have this replication of the RPE65 protein. And we just began this clinical trial in children. We have five children with retinal dystrophy who were treated with Luxturna. We are very happy because we have had some very good results for some of them. And I think we are just beginning the progress. There are other gene therapies going after other mutations. It's also very exciting to know that it's for the retina, but it could also be for the cornea, as we discussed Ataluren, in the cornea in aniridia. It could be this gene therapy, just jumping over the stop codon to replicate the protein. So I think we are just in the beginning area of gene therapy. But, amazingly, we can imagine having patients recover. So children who are supposed to lose vision completely now can regain vision. So this is a hope for patients. All of us, that's very, very important because there are so many diseases that cannot just be cured, unfortunately, with more conventional pharmaceutical or surgical methods. Gene therapy represents the cutting edge of science, and it's got to be applied with the full scientific basis, of course. For sure!

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