Bioengineered Stem Cell-Derived Cone Photoreceptor Therapy

Summary

Drug therapy only slows the progression of disease, but does not represent a regenerative approach to macular degeneration treatment. We will use cell regenerative techniques to produce large quantities of cone photoreceptors for transplantation directly into the retina. Cones are the cells responsible for high-resolution/color vision and are lost in disease. We will take advantage of natural biomaterials as vehicles to deliver cells to the eye, to increase their survival and improve their function after transplantation.

Project Details

We are focused on the goal of producing large quantities of cone photoreceptors for transplantation directly into the retina. Cones are the cells responsible for high-resolution/color vision and are lost in AMD.

Our first aim is to use regenerative techniques to derive cells that behave like the stem cells that grow in human eyes (“retinal stem cells”). We will treat these mass-produced retinal stem cells with specific chemical factors to produce cone photoreceptors with high purity and high efficiency. We will investigate the biologic mechanisms at play during this process to better understand how to produce these therapeutically important cells. In the second phase of our project, we will take advantage of natural biomaterials as vehicles to deliver cells to the eye. In doing so, we will look to increase their survival and improve retinal function after transplantation.

Drug therapy only slows the progression of disease, but does not represent a regenerative approach to macular degeneration treatment. We foresee that, given the state of the stem cell field, early success and key findings from this project are poised to shift the paradigm of therapeutic cell sources for the treatment of AMD toward the targeted production of specific, clinically-relevant cell types to replace those lost in disease. From a clinical perspective, improved stem cell-derived cone transplantation will show that the adult retina can be repopulated with transplanted photoreceptors, and can be a viable approach to treat patients suffering from AMD.