SUMMARY
Research in the laboratory of Alan D. Marmorstein, Ph.D., is driven by the goal of restoring or preventing vision loss due to retinal degenerative diseases. The research approach to vision loss is done through regenerative medicine. He uses a team-based method and works with vitreoretinal surgeons and biomedical engineers.
Dr. Marmorstein has three main lines of research. The first seeks to understand the pathogenesis of sub-retinal pigment epithelium (RPE) deposits that are characteristic of age-related macular degeneration. These deposits interfere with the exchange of nutrients and metabolites between the retina and the choriocapillaris. The second is developing a cell therapy to replace lost, damaged or dysfunctional retinal pigment epithelial cells for treatment of age-related and inherited macular degeneration using induced pluripotent stem cells. The third is to improve methods for gene therapy delivery in people with retinal disease.
Focus areas
- Inherited macular degeneration. Studies in Dr. Marmorstein's lab involve using animal models and stem cells to understand the pathogenesis of inherited macular degenerative diseases such as Best disease and Doyne honeycomb retinal dystrophy.
- Cell therapy. He develops an allogeneic-induced pluripotent stem cell-derived RPE transplant system for treatment of age-related and inherited macular degeneration. This includes cells, hydrogel scaffolds, storage and surgical delivery tools.
- Gene therapy. Dr. Marmorstein adapts the hydrogel scaffolds developed for RPE transplantation for use in gene therapy.
Significance to patient care
For most forms of macular and retinal degenerative diseases, there are no cures or therapies that can prevent loss of vision. Dr. Marmorstein provides new choices for treatment of age-related macular degeneration and inherited retinal diseases such as Best disease and Doyne honeycomb retinal dystrophy.
He is accomplishing this by creating cutting-edge cell and gene therapy treatments that replace lost cells or rescue function in cells that are no longer able to support vision. This makes it possible to restore vision in people who have lost sight and prevent loss of vision in those at risk.
Professional highlights
- Member, editorial board, PLOS ONE, 2023-present.
- Associate editor, Frontiers in Cell and Developmental Biology, 2021-present.
- Associate editor, Journal of Ocular Pharmacology and Therapeutics, 2012-present.
- Member, editorial review board, Molecular Vision, 2010-present.
- First-Round Award, Mayo Clinic President's Discovery Translation Program, "Curing Macular Degeneration With Induced Pluripotent Stem-Cell-Derived Retinal Pigment Epithelial Cells," Mayo Clinic, 2020.
- Fellow, Association for Research in Vision and Ophthalmology, 2010.