Year 1

The broad goal of this project is to develop a stem-cell derived replacement for RPE cells in the eye that die in patients with age-related macular degeneration (AMD). Since RPE cells are often genetically defective in AMD, we will correct this defect in the stem-cell derived RPE cells before transplanting them into patients. These transplanted cells will express specific proteins that will protect them from being attacked by the innate immune system. During the first year of funding we made excellent progress toward this goal. We learned how to generate RPE cells from mouse and human stem cells of various sources. We prepared all the DNA constructs that will be required to protect the transplanted RPE cells from attack by the innate immune system. We also developed a new approach for injecting RPE cells into the correct part of the mouse eye (subretinal space) without damaging other parts of the retina. We are enthusiastic to continue our work on this exciting project.