Postmortem Retinal Structural and Metabolic Analysis After Human Embryonic Stem Cell-derived Retinal Pigment Epithelium Transplantation in a Patient With Stargardt Disease.

Purpose: Stargardt disease is an inherited form of retinal degeneration characterized by early-onset central vision loss. This report describes the long-term retinal histologic characteristics of a patient with Stargardt disease after human embryonic stem cell-derived retinal pigment epithelium (hESC-RPE) transplantation.

Methods

Eyes were obtained postmortem from an 80-year-old legally blind male patient with Stargardt disease. The patient had previously undergone hESC-RPE transplantation of his left eye, and the right eye (untransplanted) served as a control. Fluorescence lifetime imaging microscopy was used to study retinal structure and metabolic activity. Staining of the retina for lipofuscin was performed using the Armed Forces Institute of Pathology method.

Results

Metabolic analysis showed that the macular area had more oxidative phosphorylation relative to the mid-peripheral retina, in both the transplanted and untransplanted eye. Melanin-laden RPE cells were detected in the transplanted eye, which correlated with a pigment band present on fundus images post-hESC-RPE transplantation.

Conclusions

This case description illustrates the long-term survival of subretinal hESC-RPE cells post-transplantation in a patient with Stargardt disease.