Research in the laboratory of Alan D. Marmorstein, Ph.D., is driven by the goal of restoring or preventing vision loss due to common and inherited eye diseases and trauma. Dr. Marmorstein studies how mutations in certain genes cause inherited forms of macular degeneration, how pressure is regulated within the eye and how some organisms regenerate their eyes in response to injury. The lab uses this information to identify and test new therapies, such as stem cells, for diseases like macular degeneration and glaucoma.
- Best disease. Dr. Marmorstein's research team investigates the use of stem cells to model and cure Best disease and other diseases caused by mutations in the gene BEST1.
- Inherited macular degeneration. Studies in Dr. Marmorstein's lab involve using stem cells to understand the pathogenesis of other inherited macular degenerative diseases.
- Fluid regulation in the eye. Research is directed at understanding how the eye controls the rate of fluid drainage from the anterior chamber.
- Regenerative medicine. Dr. Marmorstein is interested in understanding how planaria, a type of flatworm made up of 20 percent stem cells, regenerates its eyes. His goal is to be able to program human stem cells to do the same.
Significance to patient care
For most forms of macular and retinal degenerative disease, or severe ocular trauma, there is currently no cure or therapy that can prevent loss of vision. Dr. Marmorstein and his team have three goals. The first is to provide options for treatment of inherited retinal diseases, such as Best disease, that currently have no treatment options. The second is to add new drugs that target previously unrecognized targets to the arsenal available for lowering intraocular pressure, the only strategy that has proven successful in slowing vision loss due to glaucoma. The third and final goal is to provide the option of regenerating eye tissue, or even whole eyes, using stem cells. This option would make it possible to restore vision in people who have lost it and prevent loss of vision in those at risk.
- First-Round Award Winner, Mayo Clinic President’s Discovery Translation Program, "Curing Macular Degeneration With Induced Pluripotent Stem-Cell-Derived Retinal Pigment Epithelial Cells," 2020
- Associate editor, Journal of Ocular Pharmacology and Therapeutics, 2012-present
- Editorial review board member, Molecular Vision, 2010-present
- Fellow, Association for Research in Vision and Ophthalmology, inducted 2010