Retinitis pigmentosa is an inherited degenerative condition that destroys photoreceptors in the retina and can eventually lead to irreversible blindness. In a clinical trial first, an international research team has demonstrated that optogenetic therapy is safe to use and shows potential for restoring visual function in patients with late stages of this disorder.
In a first-in-human study in 2021, the collaboration – led by José-Alain Sahel from the University of Pittsburgh School of Medicine and Botond Roska from the Institute of Molecular and Clinical Ophthalmology Basel (IOB) – demonstrated that optogenetic therapy led to partial recovery of visual function in a blind patient with retinitis pigmentosa. This latest work, reported in the New England Journal of Medicine, expands the cohort to 10 participants.
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