Modelling and rescue of RP2 Retinitis Pigmentosa using iPSC Derived Retinal Organoids
Amelia Lane, Katarina Jovanovic, Ciara Shortall, Daniele Ottaviani, Anna Brugulat-Panés, Nele Schwarz, Rosellina Guarascio, Matthew John Hayes, Arpad Palfi, Naomi Chadderton, Gwyneth Jane Farrar, Alison J. Hardcastle, Michael E. Cheetham
This study uses iPSC-derived retinal organoids to model RP2 retinitis pigmentosa and shows that AAV-mediated gene augmentation can rescue photoreceptor degeneration.
Stem Cell Reports · Jun 11, 2020Peer-reviewedNeuroscience