CORDIS Project
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This project focuses on developing advanced gene delivery methods to treat inherited retinal diseases that cause blindness. By enhancing the capacity of adeno-associated virus vectors or improving lentiviral and adenoviral vectors for photoreceptor targeting, the project aims to enable effective gene therapy for condit…
Inherited retinal diseases (IRDs) cause blindness in over 200,000 individuals in Europe.
The majority are due to mutations in genes expressed in photoreceptors (PR) in the retina.
We have recently demonstrated the safety and efficacy of gene therapy for IRDs in patients with Leber congenital amaurosis (LCA).
One of the major limitations to extend this clinical success to other blinding conditions is that many are caused by mutations in genes with large coding sequences that exceed the cargo capa…
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