The safety and efficacy of hESC-derived RPE cells was assessed in several preclinical studies.
The long-term functional rescue using hESC-derived RPE has been shown in both the Royal College of Surgeons (RCS) rat, an animal model for retinal degeneration, and Elov14 mouse, an animal model for Stargardt's disease.
After introduction to the subretinal space of RCS rats, the hESC-derived RPE cells survived for more than 8 months without evidence of pathological consequences. hESC-RPE cells appear to appropriately engraft in the correct structures of the eye and also rescued visual functions in a dose-dependent fashion: with increased cell concentrations from 5,000 to 50,000. The improvement in functional rescue was measured with both visual acuity and luminance threshold response.
The Good Laboratory Practice (GMP)-compliant study was carried out in the NIH III immune-deficient mouse model. Long-term data that included the life-span of the animals) showed no gross or microscopic evidence of teratoma/ tumor formation after subretinal hESC-RPE transplantation.
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hESC-derived RPE cell treatment in animal model of retinal dystrophy has slowed the natural progression of the disease by promoting photoreceptor survival.
Visual performance improved by up to 100% over untreated control without evidence of overt pathological response in the host retina. The cells did not undergo tumerous transformation after long-term transplantation.