• Delivering innovative stem cell therapy to slow vision loss

Alan Marmorstein, PhD a researcher looking through a microscope in a laboratory

The regenerative eye scientist, Alan Marmorstein, Ph.D., is drawn to research by the need to find new cures for diseases. As an aspiring researcher, he never envisioned his investigative journey would take him down the path of testing ways to prevent or restore loss of eyesight. However, a post-doctoral lab assignment, a National Eye Institutes grant and inspiration from retina surgeons presented him the opportunity to research disorders of the eye. It launched him on a transformative career trajectory.

“Initially, I was disappointed, because I was interested in pursuing a different line, says Dr. Marmorstein. “After I started working in the lab, I fell in love with the eye as a system. The eye is a unique organ in the body that you can literally look into. You can monitor what you are doing and see the effect of your research much easier than in a lot of other places in the body. It’s very easy to figure out if something can see.”

At Mayo Clinic, Dr. Marmorstein’s research to prevent blindness includes delivery of stem cell replacement therapy to slow or stop progression of diseases that rob people of their central field of vision. Mayo Clinic Center for Regenerative Medicine supports Dr. Mamorstein’s research as part of its mission to empower discovery, advance the practice and develop new cures that address unmet patient needs.

Research focus

Dr. Marmorstein’s research focuses on age-related macular degeneration and an inherited form called Best disease, which typically afflicts people under the age of 65. Macular degeneration is a loss of central vision and a leading cause of blindness in older adults. It affects more than 10 million Americans, according to the American Macular Degeneration Foundation. While there are eye injections to ease symptoms of some forms of macular degeneration, researchers are trying to solve the puzzle of how to stop the progression and ultimately cure the disease.

“The goal of our research is to keep as many people as we can from losing their central vision. Very likely any future treatments will involve a surgical intervention. We are seeking a therapy that’s off the shelf, meaning that patients can be treated readily. Our hope is that once someone is diagnosed we could immediately offer a date for surgery,” says Dr. Marmorstein.

In dry macular degeneration, the center of the retina deteriorates. With wet macular degeneration, leaky blood vessels under the retina cause blurred vision. Little is known about the cause of age-related macular degeneration. Best disease is linked to a genetic mutation affecting  retinal pigment epithelial (RPE) cells, which are the layer of cells that support the health and function of light sensing cells in the retina. RPE cells are essential to maintaining vision.

Read the rest of the article on the Center for Regenerative Medicine blog.


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