A surgeon injects a sheet of lab-grown eye cells behind a patient’s retina to halt the blindness caused by age-related macular degeneration (AMD). AMD destroys the retinal pigment epithelium (RPE), a cell layer that keeps the eye’s light-sensing photoreceptors alive. Without it, central vision—needed for reading, recognising faces, and driving—fades to legal blindness. Currently, no treatment restores these lost RPE cells. This project aims to change that by growing functional RPE monolayers from human embryonic stem cells, then transplanting them into the eye using a minimally invasive procedure. The team has two decades of experience in retinal cell transplantation and stem cell research. Their immediate goal is to complete preclinical tests that satisfy FDA requirements, paving the way for a clinical trial in patients at risk of vision loss from AMD. If successful, this approach could prevent the irreversible death of photoreceptors, preserving sight for hundreds of thousands of people. The work is applied regenerative medicine, not fundamental science—it targets a specific clinical pathway with a clear regulatory endpoint.
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Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being of Californians. It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-related macular degeneration (AMD), the most common cause of retinal degeneration diseases in the elderly. AMD is a progressive ocular disease of the part of the retina, called the macula, which enables people to read, visualize faces, and drive. The disease initially causes distortion in central vision, and eventually leads to legal blindness. A layer of cells at the back of the eye called the retinal pigment epithelium (RPE), provide support, protection, and nutrition to the light sensitive cells of the retina; the photoreceptors which consist of rods and cones . The dysfunction and/or loss of these RPE cells play a critical role in the loss of the PR?s and hence the blindness in AMD. Effective treatment could be achieved by proper replacement of damaged RPE and retinal cells with healthy ones. More specifically, the regenerated and restored RPE layer would prevent the irreversible loss of the PR?s. However, the lack of a feasible approach to restore the RPE cells has prevented the realization of a potential therapy. Recent advances in knowledge and technology of human embryonic stem (hES) cells brings new hope for the development of cell replacement treatment. hES cells are capable of unlimited self-replication and production of different cell types. RPE cells derived from hES cells are a potentially unlimited and robust source for regenerating RPE. We hypothesize that the dysfunction and/or loss of RPE can be overcome by regenerating and restoring the RPE through the transplantation of functionally polarized RPE monolayers derived from hES cells. Such RPE cells derived from hES can then be transplanted into the eye, using minimally invasive surgical procedures saving the PR from dying. Our group is composed of unique multidisciplinary members who collectively have more than two decades of experience in efforts to restore sight to the blind as well as retinal cell transplantation and stem cell research. Our plan for this grant is to use our expertise and infrastructure to show to the FDA the success of our preclinical tests using hES derived RPE cells in order to get approval to conduct a clinical trial in patients at risk of vision loss due to AMD. Limit Public
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