GMP-compliant Human RPE Cells Derived from Embryonic Stem Cell Lines Rescue Visual Function In a Rat Model for Photoreceptor Degeneration

B. Lu1 ; S. Wang1; S. Girman1; P.J. Francis1; J. Lund2; C. Malcuit3; L. Vilner3; L. Lemieux3; R. Lanza3; P. Huertas3,4; R.D. Lund1,2.
1. Casey Eye Institute, Dept of Ophthalmology, OHSU, Portland, OR. USA; 2. Moran Eye Center; 3. ACT; 4. Harvard-MIT.

Abstract

Certain retinal diseases are characterized by degeneration of the retinal pigment epithelium (RPE) which in turn results in photoreceptor loss. Examples include Stargardt macular dystrophy in humans and the genetically-determined dystrophy in the Royal College of Surgeons (RCS) rat. Such a process may also play a role in age related macular degeneration, which is responsible for compromised vision in more than 9 million people in the US alone. We investigated whether a GMP human embryonic stem cell-derived RPE (hESRPE) cell line could rescue visual function in the dystrophic RCS rat. GMP-compliant hES-RPE cells (developed by Advanced Cell Technology) were injected into the subretinal space of 22 day-old (P22) RCS rats that were maintained on oral cyclosporine immunosuppression, post-operatively. Functional efficacy was tested by threshold optomotor acuity and luminance thresholds recorded from the superior colliculus. All treated eyes were compared with sham-injected and untreated eyes. Histological examination was performed after these functional assessments

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