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This pioneering open-label study examines the safety and initial efficacy of a ganglion cell-targeted optogenetic gene therapy using an adeno-associated viral vector encoding ChrimsonR in patients with advanced retinitis pigmentosa (RP). Intravitreal injection combined with the use of specialized light-stimulating goggles resulted in meaningful improvements in light sensitivity in a majority of participants. Despite some ocular adverse events, including one transient severe event, overall safety was acceptable in this small cohort, warranting further investigation.
