The New England journal of medicine
Optogenetic Therapy for Restoring Aspects of Visual Function
Por Sahel JA, Futterknecht S, Audo I y 17 más.
Contenido en inglés
Resumen
Background
Retinitis pigmentosa is an inherited degenerative retinal disease that can lead to irreversible blindness. Optogenetic therapy has shown potential for restoring visual function at late stages of the disease.
Methods
In this open-label study, we evaluated the safety of ganglion cell-directed optogenetic therapy in 10 participants with blindness due to advanced retinitis pigmentosa. Each participant received a single intravitreal injection of an adeno-associated viral (AAV) vector encoding the red-shifted channelrhodopsin ChrimsonR in the worse-seeing eye. The primary outcome was safety. Secondary outcomes included changes in light sensitivity as measured by full-field stimulus threshold (FST) testing conducted with the untreated eye patched and with the use of light-stimulating goggles designed to activate ChrimsonR. The definition of a clinically meaningful improvement in the FST (based on evidence from the literature and not prespecified in the protocol) was a decrease of at least 0.6 log units (i.e., an increase in light sensitivity by a factor of approximately 4).