Nature Communications · 2016 · 319 citations · 49 references
MedicineCell BiologyOphthalmologyCell TherapyOcular DiseaseVisual DisordersPhotoreceptor PhysiologyRetinal DevelopmentPhotoreceptor BiologyPhotoreceptor CellsPhotoreceptor RegenerationVisual Function Loss
Photoreceptor replacement by transplantation is proposed as a treatment for blindness. Transplantation of healthy photoreceptor precursor cells into diseased murine eyes leads to the presence of functional photoreceptors within host retinae that express an array of donor-specific proteins. The resulting improvement in visual function was understood to be due to donor cells integrating within host retinae. Here, however, we show that while integration occurs the majority of donor-reporter-labelled cells in the host arises as a result of material transfer between donor and host photoreceptors. Material transfer does not involve permanent donor-host nuclear or cell-cell fusion, or the uptake of free protein or nucleic acid from the extracellular environment. Instead, RNA and/or protein are exchanged between donor and host cells in vivo. These data require a re-evaluation of the mechanisms underlying rescue by photoreceptor transplantation and raise the possibility of material transfer as a strategy for the treatment of retinal disorders.
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Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text
Medicine, Genetics, Ophthalmology +17
Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
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Retinal repair by transplantation of photoreceptor precursors
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