CORDIS Project
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This project focuses on advancing gene therapy for retinal diseases by creating non-human primate models of retinal degeneration. It aims to develop novel adeno-associated viral vectors for effective gene delivery, facilitating the testing of therapies aimed at restoring vision.
Gene therapy using adeno-associated viral (AAV) vectors has shown early promise in clinical trials.
The therapeutic transgene cassette can be packaged in different AAV capsid pseudotypes, each having a unique transduction profile.
At present, AAV capsid serotype selection for a specific clinical trial is based on effectiveness in small animal models.
We (and others) have shown substantial progress in improving gene therapy for eye diseases in rodents.
However, small animal studies are not often…
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