Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)
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 retinal photoreceptors (PR's), light sensitive 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 human hES 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 can then be transplanted into the eye, using customized surgical procedures and instruments 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 stage of this grant is to use our expertise and infrastructure to study and ultimately show the success of using hES derived RPE cells to the FDA in order to get approval to conduct a clinical trial in patients blind due to AMD.
University of Southern California
Disease Team Planning