Project Title: Culture and Transplantation of Human Retinal Spheres Principal Investigator: David Gamm, MD, PhD Inherited and acquired eye diseases that target the retina and retinal pigment epithelium (RPE) are a significant cause of vision loss in children and adults. Progressive vision loss, in turn, can exacerbate or lead to more global developmental disabilities and reduce functional capacity on numerous levels. The existence of distinct stem cell populations in many human tissues, including brain, has raised interest in the therapeutic applications of stem cell biology. Innovative methods of growing stem cells from retina or RPE may produce large populations of transplantable cells capable of preventing or repairing retinal degenerative diseases. Therefore, we have developed a culture system to grow free-floating spherical collections of primitive retinal and RPE cells from human embryonic eye specimens. Cells prompted to migrate outward from these spheres mature and adopt different shapes, pigmentation patterns and identifying characteristics. Furthermore, they can express lentiviral gene constructs, and thus may be useful in ex vivo gene therapy strategies. Based on these findings, we aim to 1) further characterize stem cells from prenatal and adult human eyes, 2) investigate their response to different culture treatments in an effort to specify cell fate, and 3) examine their capacity to preserve or replace retinal cells in animal models of retinal degeneration. By understanding the behavior of these cells in the culture dish and in living animals, we hope to optimize strategies to delay or reverse the effects of potentially devastating human diseases of the visual system.

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