Slush Science Pitching Finals 2017: Ivana Trapani
summary
Claims a gene‑editing therapy for Stargardt disease that uses a modified, non‑replicating viral vector to deliver molecular scissors that cut out the mutated region in retinal cells, aiming to restore function. Builds and tests on 3D human retinal organoids derived from patients, shows correction at the cellular level in vitro, and frames impact for tens of thousands affected (cites ~75,000). Focuses on single‑point mutation correction and preclinical efficacy data.
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