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Development of safe and efficient aav vectors for retinal gene therapy

Abstract : Vision is our most cherished sense and its loss is a feared handicap. A highly diverse and complex array of inherited retinal degenerations leads to irreversible vision loss. Today, there is no cure for such disorders. However, in the last decade, many gene therapies entered clinical trials offering hope for the treatment of inherited retinal degenerations. In this thesis, we explored the contribution of viral vectors within the general context of retinal gene therapy. We focused on optimization of viral vectors for mutation-independent gene therapies broadly applicable across rod-cone dystrophies. We carefully designed vectors for targeting cones and studied their translational potential for optogenetic activation of cones in several relevant model systems.
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Submitted on : Monday, August 31, 2020 - 12:33:53 PM
Last modification on : Thursday, September 3, 2020 - 12:04:04 PM


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  • HAL Id : tel-02926062, version 1


Hanen Khabou. Development of safe and efficient aav vectors for retinal gene therapy. Neurons and Cognition [q-bio.NC]. Sorbonne Université, 2018. English. ⟨NNT : 2018SORUS460⟩. ⟨tel-02926062⟩



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