WP8 will integrate the gene editing and transfer technologies developed by the consortium to tackle Retinitis Pigmentosa caused by gain-of-function or dominant negative mutations in the Rho gene in model mice.
Specific WP’s objectives:
To test the therapeutic efficacy of AAV-mediated homology-independent targeted integration (HITI) in animal models of retinal degenerations
Development of non-viral vesicles (VEsiCas) for the delivery of CRISPR/Cas9 in the retina
Task 8.1: AAV-mediated HITI in animal models of retinal degeneration
Task 8.2: Development of VEsiCas for mRHO editing in retinal degeneration
WP leader: Alberto Auricchio (FTELE)
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