“There is no cure for LCA yet,” said Laura. “But because of Dr. Stone, we believe that there can be—and that it can happen in Molly’s lifetime.”

When Molly Troxel was seven years old, she wrote this wish in a note to Dr. Edwin Stone, the Seamans-Hauser Chair in Molecular Ophthalmology at University of Iowa Hospitals and Clinics. Dr. Stone had already been her eye doctor for six years.

Molly is nine now. She lives with her mom, dad, and two sisters in Omaha, Nebraska, and she loves drawing, playing with Barbies, riding her bike, and Justin Bieber…just like any other fourth-grader.

But Molly has a rare inherited eye disease called Leber’s congenital amaurosis (LCA), a condition that limits her vision to 20/200, with her glasses on.
LCA appears at birth or in the first few months of life and affects around 1 in 80,000 of the population.
When she wears contacts, her vision improves to 20/100.

“Everybody knows that Molly is visually impaired except Molly,” laughed her mom, Laura Troxel.

Molly comes to Iowa City to see Dr. Stone once a year, and because of the research that he and his team are pursuing in the Iowa Institute for Vision Research (IVR), Molly and her family have real hope that her vision may be restored someday.

“There is no cure for LCA yet,” said Laura. “But because of Dr. Stone, we believe that there can be—and that it can happen in Molly’s lifetime.”

LCA, caused by multiple genes, has been successfully treated with gene therapy. In fact, doctors in the UI Laboratory for Gene Discovery have pinpointed the gene that causes Molly’s type of LCA—a breakthrough that brings them closer than ever to being able to restore her vision.

“I’ll never forget the first time we brought Molly to the UI. She was barely a year old,” Laura continued. “Dr. Stone told us the story of a dog named Lancelot with LCA, who had regained sight as a result of gene therapy—and then he assured us that he and his team were doing everything in their power to make sure that human patients like Molly would have access to effective gene therapy, too.”

Molly’s dad agrees. “At first we were afraid, leery, and not sure what to do,” said Ryan Troxel. “Dr. Stone and the other doctors and nurses not only made time for us and reassured us, but they have already made amazing progress in their research in the short time Molly has been a patient here.”

Molly’s note has been scanned, copied, and shared multiple times—across the Department of Ophthalmology and Visual Sciences, within the labs of the IVR, in PowerPoint presentations by the department, and even in the hallways of the UI Foundation, where private funds are raised to support these areas—because it so eloquently expresses what inspires Dr. Stone and his fellow UI visual-sciences colleagues to keep pushing their work to the next level.

These top-tier physicians and researchers have won countless awards and continue to make innumerable advances toward their common goal of preventing and curing blinding eye diseases for good. They are among the best in the world, and any number of them could actually be in the running for a Nobel Prize some day.

But they don’t do it for the honors and accolades. Their breakthroughs are inspired by the wish of a little girl—and every patient like her—who simply wants to see.