Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)

点击翻译按钮获取中文摘要

Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being ofCalifornians. It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-relatedmacular degeneration (AMD), the most common cause of retinal degeneration diseases in the elderly. AMD is a progressiveocular disease of the part of the retina, called the macula, which enables people to read, visualize faces, and drive. The diseaseinitially 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 tothe light sensitive cells of the retina; the photoreceptors which consist of rods and cones . The dysfunction and/or loss of theseRPE cells play a critical role in the loss of the PR?s and hence the blindness in AMD. Effective treatment could be achieved byproper replacement of damaged RPE and retinal cells with healthy ones. More specifically, the regenerated and restored RPElayer would prevent the irreversible loss of the PR?s. However, the lack of a feasible approach to restore the RPE cells hasprevented the realization of a potential therapy.Recent advances in knowledge and technology of human embryonic stem (hES) cells brings new hope for the development ofcell replacement treatment. hES cells are capable of unlimited self-replication and production of different cell types. RPE cellsderived 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 thetransplantation of functionally polarized RPE monolayers derived from hES cells. Such RPE cells derived from hES can then betransplanted 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 inefforts to restore sight to the blind as well as retinal cell transplantation and stem cell research. Our plan for this grant is to useour expertise and infrastructure to show to the FDA the success of our preclinical tests using hES derived RPE cells in order to getapproval to conduct a clinical trial in patients at risk of vision loss due to AMD.Limit Public