Among the institute’s primary activities will be vision research, and among those research projects will be the gene therapy work of husband-and-wife team Maureen and Jay Neitz, professors of ophthalmology, who began their collaboration 23 years ago.
“We thought that if we could better understand how the visual system works, we could help people who can’t see,” says Jay Neitz.
The Neitzes began investigating color blindness, which involves a genetic mutation that results in missing or damaged photoreceptors in the retina — those cells that distinguish red, green, and blue. The Neitzes theorized that replacing these defective genes with healthy ones might lead to normal vision. In 2007, they cured color blindness in squirrel monkeys using gene therapy.
Recognizing that the photoreceptors of the eye responsible for color are the same cells that we rely on to see in regular light, the Neitzes are also looking at gene therapy as a treatment for other vision-related conditions.
Curing color blindness served as a proof of principle. “It’s an issue of simply understanding the mechanism,” says Maureen Neitz. Now, adds Jay Neitz, “we’re at a place where this understanding can be applied.” Some of their work has suggested that it might be possible to slow down degenerative processes that lead to blindness and nearsightedness.
In addition to replacing defective genes in photoreceptors, UW Medicine Eye Institute researchers are also attempting to replace the photoreceptors themselves to restore vision to people with retinal degeneration. Tom Reh, for example, has been looking at ways to treat retinal diseases using stem cells. Recently, a graduate student in his laboratory developed a technique for transplanting photoreceptors developed from stem cells into mice. This confirmation of a lifetime of hypotheses is gratifying, says Reh, but knowing that it works is not enough. These discoveries need to be applied to benefit patients.
And therapies for patients are in the works. Reh, Van Gelder, and their colleagues at the UW Institute for Stem Cell and Regenerative Medicine are planning for the first clinical trials using stem cells to restore vision.
Successful research like Reh’s depends, in large part, on funding — on obtaining the resources to recruit expert faculty members who can compete for grants. Large eye centers in Boston, California, London, and Wisconsin are involved in similar work, says Reh, and the creation of the eye institute will make UW Medicine more competitive in the field of eye-related research and care.
“It’s a matter of who’s going to do it first,” Reh says. “If Seattle wants to be competitive in this field, the eye institute is critical.”
Russell Van Gelder, chair of the Department of Ophthalmology, anticipates that the UW Medicine Eye Institute will make an already strong training program even more competitive. Resident training at UW Medicine takes a number of forms, including teaching sessions, such as the one, pictured above, that Van Gelder is leading. Residents also are taught in outpatient and surgical settings.