CORDIS Project
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This project focuses on developing enhanced adeno-associated virus variants for gene therapy targeting inherited retinal degenerations. It aims to improve gene delivery to the outer retina in both mice and primates, addressing challenges in translating therapies from animal models to humans.
Inherited retinal degenerations are a primary clinical focus of adeno-associated virus (AAV) mediated gene therapy.
These diseases mostly involve pathogenic mutations in the retinal pigment epithelium or photoreceptor transcripts leading to cell death and progressively lead to blindness.
Current delivery methods are efficient for gene delivery to these cells as well as other therapeutically relevant cell types in the mouse retina.
However the applicability of these vectors in human gene therapy…
UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6
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